[et_pb_section fb_built=”1″ admin_label=”Home 2 Content Pg” _builder_version=”4.16″ _module_preset=”default” background_color=”#bbe4ed” background_image=”https:\/\/epitomestaging.com\/tnc\/wp-content\/uploads\/2022\/02\/rsz-tnc-header-engagement-1000×200-1.jpg” background_size=”initial” background_position=”top_center” width_phone=”auto” width_last_edited=”off|desktop” max_width_last_edited=”off|desktop” module_alignment=”center” min_height_tablet=”300px” min_height_last_edited=”off|desktop” height=”200px” height_tablet=”154px” height_phone=”100px” height_last_edited=”on|phone” max_height_tablet=”300px” max_height_phone=”300px” max_height_last_edited=”off|desktop” custom_margin=”0px||0px||false|false” custom_padding=”0px||0px||false|false” top_divider_height_tablet=”” top_divider_height_phone=”50px” top_divider_height_last_edited=”on|phone” bottom_divider_style=”curve” bottom_divider_color=”#FFFFFF” bottom_divider_height=”70px” bottom_divider_arrangement=”above_content” bottom_divider_height_tablet=”70px” bottom_divider_height_phone=”50px” bottom_divider_height_last_edited=”on|desktop” background_last_edited=”on|tablet” background_image_phone=”https:\/\/epitomestaging.com\/tnc\/wp-content\/uploads\/2022\/02\/rsz-tnc-mobile-stars.jpg” background_enable_image_phone=”on” parallax_tablet=”off” parallax_phone=”off” background_size_tablet=”contain” background_size_phone=”initial” background_position_tablet=”top_center” background_position_phone=”top_right” background_enable_video_mp4_tablet=”off” background_enable_video_mp4_phone=”off” module_alignment_tablet=”right” module_alignment_phone=”left” module_alignment_last_edited=”off|desktop” locked=”off” global_colors_info=”{}”][et_pb_row column_structure=”1_2,1_2″ use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ _module_preset=”default” module_alignment=”center” custom_margin=”0px||||false|false” custom_padding=”0px||||false|false” global_colors_info=”{}”][et_pb_column type=”1_2″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text content_tablet=”<\/p>\n
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[\/et_pb_text][\/et_pb_column][et_pb_column type=”1_2″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ _builder_version=”4.16″ _module_preset=”default” background_enable_color=”off” custom_margin=”20px||21px||false|false” custom_padding=”0px||0px||false|false” locked=”off” global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ _module_preset=”default” custom_padding=”0px|0px|0|0px|false|false” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n
[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ _builder_version=”4.20.0″ _module_preset=”default” background_enable_color=”off” custom_margin=”20px||30px||false|false” custom_padding=”40px|0px|0px|0|false|false” hover_enabled=”0″ locked=”off” global_colors_info=”{}” sticky_enabled=”0″][et_pb_row column_structure=”1_3,1_3,1_3″ use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.27.0″ _module_preset=”default” custom_padding=”0px|0px|0px|0px|false|false” global_colors_info=”{}”][et_pb_column type=”1_3″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” custom_margin=”0px||0px||false|false” custom_padding=”0px||0px||false|false” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n
CDKL5 Deficiency Disorder > <\/a>Chopra-Amiel-Gordon Syndrome ><\/a> Down Syndrome > \u00a0 <\/a>Dravet Syndrome ><\/a>Duchenne Muscular Dystrophy ><\/a>Epilepsy ><\/a> \u00a0\u00a0 \u00a0\u00a0 \u00a0\u00a0 \u00a0\u00a0 \u00a0\u00a0 \u00a0\u00a0Friedreich’s Ataxia ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_3″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” custom_margin=”0px||0px||false|false” custom_padding=”0px||0px||false|false” global_colors_info=”{}”]<\/p>\n GRIN-Related Neurodevelopmental Disorder ><\/a>MECP2 Duplication Syndrome ><\/a>\u00a0Myasthenia Gravis ><\/a> Myotonic Dystrophy ><\/a>Nemaline Myopathy ><\/a>Phelan-McDermid Syndrome ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_3″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”0px||0px||false|false” custom_padding=”0px||0px||false|false” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n PTEN Hamartoma Syndrome ><\/a> Rett Syndrome ><\/a>Spinal Muscular Atrophy ><\/a>Sturge Weber Syndrome ><\/a> Tuberous Sclerosis Complex ><\/a>X-Linked Myotubular Myopathy ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”asd” _builder_version=”4.20.2″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ _module_preset=”default” custom_padding=”0px|0px|0|0px|false|false” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” header_2_text_color=”#000000″ global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”cags” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” locked=”off” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.16″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”cdkl5″ _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Heather Olson, MD, MS- NCT05558371<\/a><\/p>\n Patient Advocacy Group Partner: International Foundation for CDKL5 Research (IFCR)<\/a><\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Heather Olson, MD, MS – NCT05373719<\/a><\/p>\n Patient Advocacy Group Partner: LouLou Foundation <\/a><\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Christelle Achkar, MD – NCT05064878<\/a><\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Heather Olson, MD, MS – NCT06700811<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”cags” _builder_version=”4.27.5″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” locked=”off” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.16″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Maya Chopra, MBBS, FRACP \u2013 NCT05528744<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”ds” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” locked=”off” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Sabrina Sargado, MD \u2014\u00a0NCT04219280<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”dravets” _builder_version=”4.27.6″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” locked=”off” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” hover_enabled=”0″ custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}” sticky_enabled=”0″][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Christelle Achkar<\/span>, MD \u2014 NCT06872125<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”dmd” _builder_version=”4.27.5″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” locked=”off” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Partha Ghosh, MD<\/span>\u00a0\u2014 NCT06270719<\/a>\u200b<\/span><\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Partha Ghosh, MD<\/span>\u00a0\u2014 NCT07127978<\/a><\/p>\n Patient Advocacy Group Partner: Muscular Dystrophy Association<\/a>, Parent Project Muscular Dystrophy (PPMD)<\/a>\u200b<\/span><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”epi” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px|0px|40px|0px|false|false” border_width_bottom=”1px” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Alexander Rotenberg MD, PhD \u2013 NCT05944692<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”fa” _builder_version=”4.27.5″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px|0px|40px|0px|false|false” border_width_bottom=”1px” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Leslie Hayes, MD \u2013 NCT06865482<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”grin” _builder_version=”4.27.5″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.0″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”2px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.20.0″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_padding=”||0px|30px|false|false” border_style_bottom=”none” global_colors_info=”{}”]<\/p>\n PI: Christelle Achkar, MD – NCT07224581<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”mecp2″ _builder_version=”4.27.5″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.0″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”2px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.20.0″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_padding=”||0px|30px|false|false” border_style_bottom=”none” global_colors_info=”{}”]<\/p>\n PI: David Lieberman, MD, PhD – <\/span> NCT06430385<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”gmg” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px|0px|40px|0px|false|false” border_width_bottom=”1px” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Partha Ghosh, MD \u2013 NCT05644561<\/a><\/p>\n Patient Advocacy Group Parnter: Myasthenia Gravis Foundation of America (MGFA)<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”mydy” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px|0px|40px|0px|false|false” border_width_bottom=”1px” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Leslie Hayes, MD\u2014 NCT06185764<\/a><\/p>\n Patient Advocacy Group Partner: Muscular Dystrophy Association (MDA)\u00a0<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”nemy” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px|0px|40px|0px|false|false” border_width_bottom=”1px” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Leslie Hayes, MD\u2014 NCT06774703<\/a><\/p>\n Patient Advocacy Group Partners: Muscular Dystrophy Association (MDA)\u00a0<\/a>, Cure CMD<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”pms” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” min_height=”0.8px” custom_css_main_element=”float: right;” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px|0px|40px|0px|false|false” border_width_bottom=”1px” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Siddharth Srivastava, MD<\/span> \u2014 NCT06662188<\/a><\/p>\n Patient Advocacy Group Partners: Phelan McDermid Syndrome Foundation, <\/a>CureSHANK<\/a><\/p>\n PI: Mustafa Sahin, MD, PhD \u2014 NCT02461420 <\/a><\/p>\n Patient Advocacy Group Partners: Phelan McDermid Syndrome Foundation<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”phts” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_margin=”0px||||false|false” custom_padding=”30px||0px||false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_css_main_element=”float: right;” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px|0px|40px|0px|false|false” border_width_bottom=”1px” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Mustafa Sahin, MD, PhD\u00a0 |\u00a0 Natural History Study \u2014 NCT02461446 <\/a><\/p>\n Patient Advocacy Group Partners: PTEN Foundation<\/a>, PTEN Research<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”rett” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” background_enable_color=”off” custom_css_main_element=”float: right;” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.0″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: David Lieberman, MD, PhD – NCT05606614<\/a><\/span><\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: David Lieberman, MD, PhD\u00a0 |\u00a0 Interventional Therapeutic Trial \u2014 NCT05898620<\/span><\/a><\/p>\n Patient Advocacy Group Partner: NEUROGENE INC<\/a><\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: David Lieberman, MD, PhD\u2014 NCT05432349<\/a><\/p>\n Patient Advocacy Group Partner: International Rett Syndrome Foundation<\/a><\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: David Lieberman, MD, PhD\u2014NCT06338267<\/a><\/p>\n Patient Advocacy Group Partner: Rett Syndrome Research Trust (RSRT)<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”sma” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_margin=”||0px|||” custom_padding=”30px|0px|0px|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” background_enable_color=”off” custom_css_main_element=”float: right;” border_color_bottom=”#000000″ global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_color_bottom=”#000000″ border_style_bottom=”none” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”1px|||||” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Basil Darras, MD \u2013 NCT05115110<\/a><\/p>\n Patient Advocacy Group Partners: Cure SMA<\/a>, Muscular Dystrophy Association<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.0″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”1px|||||” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Basil Darras, MD \u2013 NCT06555419<\/a><\/p>\n Patient Advocacy Group Partners: Cure SMA<\/a>, Muscular Dystrophy Association<\/a><\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_margin=”1px|||||” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Leslie Hayes, MD \u2013 NCT05335876<\/a><\/p>\n Patient Advocacy Group Partners: Cure SMA<\/a>, Muscular Dystrophy Association<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”sws” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_color_bottom=”#000000″ border_style_bottom=”none” locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: John McLaren, MD<\/span> \u00a0|\u00a0 Natural History Study \u2014 NCT04717427 <\/a><\/p>\n Patient Advocacy Group Partner: Sturge-Weber Foundation<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.0″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.27.5″ _module_preset=”default” custom_margin=”1px|||||” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: John McLaren, MD<\/span> \u2013NCT04517565<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”tsc” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_css_main_element=”float: right;” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Mustafa Sahin MD, PhD\u00a0 |\u00a0 Clinical Drug Trial \u2014 NCT05104983 <\/a><\/p>\n Patient Advocacy Group Partner: TSC Alliance<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_style_all=”none” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Mustafa Sahin MD, PhD\u00a0 |\u00a0 Natural History Study \u2014 NCT02461459 <\/a><\/p>\n Patient Advocacy Group Partner: TSC Alliance<\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ module_id=”xlmymy” _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_padding=”30px|0px|0|0|false|false” global_colors_info=”{}”][et_pb_row column_structure=”3_4,1_4″ _builder_version=”4.16″ _module_preset=”default” custom_padding=”||0px||false|false” custom_css_main_element=”display: flex;” locked=”off” global_colors_info=”{}”][et_pb_column type=”3_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}”]<\/p>\n [\/et_pb_text][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” background_enable_color=”off” custom_css_main_element=”float: right;” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”]<\/p>\n Learn More ><\/a><\/p>\n [\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”2″ _builder_version=”4.20.1″ _module_preset=”default” custom_margin=”||||false|false” custom_padding=”0px||40px||false|false” border_width_bottom=”1px” border_color_bottom=”#000000″ locked=”off” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\n PI: Leslie Hayes, MD \u2013 NCT06581146<\/a><\/p>\n Patient Advocacy Group Partner: Cure CMD<\/a>, Muscular Dystrophy Association (MDA)<\/a>, Joshua Frase Foundation<\/a>\u00a0<\/p>\n [\/et_pb_text][et_pb_text _builder_version=”4.27.6″ _module_preset=”default” custom_padding=”||0px|30px|false|false” global_colors_info=”{}”]<\/p>\nActive TNC Clinical Research Studies<\/h2>\n
Accelerating Clinical Trial Readiness Innovations for Monogenic Neurodevelopmental Disorders (ACTION) Initiative<\/h2>\n
CDKL5 Deficiency Disorder<\/h2>\n
International CDKL5 Clinical Research Network\u2019s Clinical Trial Readiness Study<\/h3>\n
Endpoint Enabling Study of Cyclin-dependent kinase-like 5 (CDKL5) Deficiency Disorder (CANDID)<\/h3>\n
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study to Examine the Efficacy and Safety of ZX008 in Subjects with CDKL5 Deficiency Disorder Followed by an Open-Label Extension<\/h3>\n
Phase 1 Study of Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies<\/span><\/h3>\n
Chopra-Amiel-Gordon Syndrome (CAGS)<\/h2>\n
Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome<\/h3>\n
Down Syndrome<\/h2>\n
Treatment Efficacy and Safety of ADHD Medication in Down Syndrome \u2013 TEAM-DS
Down Syndrome<\/h3>\nDravet Syndrome<\/h2>\n
EMPEROR: A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel Group, Phase 3 Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen (STK-001) in Patients With Dravet Syndrome<\/span><\/h3>\n
Duchenne Muscular Dystrophy (DMD)<\/h2>\n
A Long-Term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants with Duchnne Muscular Dystrophy under Conditions of Routine Clinical Practice<\/span><\/h3>\n
A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients with Duchenne Muscular Dystrophy<\/span><\/h3>\n
Epilepsy<\/h2>\n
Epilepsy Seizure Detection with Innovative Tripolar EEG (tEEG)<\/span><\/h3>\n
Friedreich’s Ataxia (FA)<\/h2>\n
Characteristics and Clinical Course of Disease In Participants with Cardiomyopathy Associated With Friedreich Ataxia<\/span><\/span><\/h3>\n
GRIN-related Neurodevelopmental Disorder<\/h2>\n
A Multinational, Multicenter Study With an Open-Label Phase 1b and a Randomized, Double-Blind, Placebo-Controlled Phase 3 Followed by an Open-Label Extension to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Radiprodil in Participants With GRIN-Related Neurodevelopmental Disorder<\/h3>\n
MECP2 Duplication Syndrome<\/h2>\n
A Phase 1-2, Double-Blind, Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients With MECP2 Duplication Syndrome<\/h3>\n
Myasthenia Gravis<\/h2>\n
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)<\/h3>\n
Myotonic Dystrophy<\/h2>\n
A Phase 1\/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type 1 (DM1) (Galileo)<\/h3>\n
Nemaline Myopathy<\/h2>\n
Nemaline Myopathy natural history study – Assessing Individuals with Nemaline Myopathy (NM-CTRN)<\/h3>\n
Phelan-McDermid Syndrome (PMS)<\/h2>\n
A Phase 1\/2, Multicenter, Open-Label, Dose-Escalation, Safety, Tolerability, and Clinical Activity Study of a Single Dose of JAG201 Gene Therapy Delivered Via Intracerebroventricular Administration in Participants With SHANK3 Haploinsufficiency<\/span><\/h3>\n
Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome<\/h3>\n
PTEN Hamartoma Syndrome (PHTS)<\/h2>\n
Natural History Study of Individuals With Autism and Germline Heterozygous PTEN Mutations<\/h3>\n
Rett Syndrome<\/h2>\n
An Open-label Phase 1\/2\/3 Study Consisting of a Phase 1\/2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome<\/h3>\n
Neurogene- A Phase 1\/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Pediatric Subjects with Rett Syndrome<\/h3>\n
IRSF Rett Syndrome Observational Registry<\/h3>\n
Validating Innovative Biosensors for Rett Autonomic Symptom Tracking (VIBRANT)<\/h3>\n
Spinal Muscular Atrophy (SMA)<\/h2>\n
A 2 part, seamless, multi-center, randomized, placebo-controlled, double blind study to investigate the safety, tolerability, pharmokinetics, pharmodynamics, and efficacy of RO7204239 in combination with Risdiplam (RO7034067) in ambulant patients with spinal muscular atrophy. (MANATEE)<\/h3>\n
An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered via the ThecaFlex DRx\u2122 System (PIERRE-PK)<\/h3>\n
AVXS long term f\/u–A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 (SPECTRUM)<\/h3>\n
Sturge-Weber Syndrome<\/h2>\n
Integrated Longitudinal Studies to Identify Biomarkers and Therapeutic Strategies for Sturge-Weber Syndrome<\/h3>\n
MRI Biomarkers in Patients with Sturge Weber Syndrome<\/h3>\n
Tuberous Sclerosis Complex (TSC)<\/h2>\n
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)<\/h3>\n
Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)<\/h3>\n
X-Linked Myotubular Myopathy<\/h2>\n
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition (EXCEL)<\/h3>\n
A Phase 1\/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants with Invasive Ventilator-dependent X-linked Myotubular Myopathy<\/span><\/h3>\n