A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study. Cohort 1 will include participants 4 to <7 years of age. Cohort 2 will include participants 7 to <12 years of age. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

开始日期2024-05-06

申办/合作机构

Solid Biosciences, Inc.

100 项与 SGT-003 相关的临床结果

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100 项与 SGT-003 相关的转化医学

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100 项与 SGT-003 相关的专利（医药）

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项与 SGT-003 相关的新闻（医药）

2024-09-23

·GlobeNewswire-Health Care

Solid Biosciences to Participate at Chardan’s 8th Annual Genetic Medicines Conference

CHARLESTOWN, Mass., Sept. 23, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at Chardan’s 8th Annual Genetic Medicines Conference on Monday, September 30, 2024, at 11:00 am ET. A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page. Institutional investors interested in meeting with management during the conferences may reach out to their Chardan representatives. About Solid BiosciencesSolid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Solid Biosciences Investor Contact:Nicole AndersonDirector, Investor Relations and Corporate CommunicationsSolid Biosciences Inc.investors@solidbio.com Media Contact:Glenn SilverFINN Partnersglenn.silver@finnpartners.com

基因疗法

2024-09-12

·GlobeNewswire-Health Care

Solid Biosciences to Participate at the 2024 Cantor Global Healthcare Conference

CHARLESTOWN, Mass., Sept. 12, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at the 2024 Cantor Global Healthcare Conference on Wednesday, September 18, 2024, at 10:20 am ET. A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page. Institutional investors interested in meeting with management during the conferences may reach out to their Cantor Fitzgerald representatives. About Solid BiosciencesSolid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Solid Biosciences Investor Contact:Nicole AndersonDirector, Investor Relations and Corporate CommunicationsSolid Biosciences Inc.investors@solidbio.com Media Contact:Glenn SilverFINN Partnersglenn.silver@finnpartners.com

基因疗法

2024-09-04

·GlobeNewswire-Health Care

Solid Biosciences to Participate at Upcoming Investor Conferences

CHARLESTOWN, Mass., Sept. 04, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced participation in the following investor conferences: H.C. Wainwright 26th Annual Global investment Conference – New York, NYBo Cumbo, President and CEO, will participate in a fireside chat on Tuesday, September 10, 2024, at 9:30 am ET. Baird 2024 Global Healthcare Conference – New York, NYMr. Cumbo will present on Wednesday, September 11, 2024, at 12:50 pm ET. A live webcast of the H.C. Wainwright fireside chat will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page. Institutional investors interested in meeting with management during the conferences may reach out to their H.C. Wainwright or Baird representatives. About Solid BiosciencesSolid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Solid Biosciences Investor Contact:Nicole AndersonDirector, Investor Relations and Corporate CommunicationsSolid Biosciences Inc.investors@solidbio.com Media Contact:Glenn SilverFINN Partnersglenn.silver@finnpartners.com

基因疗法

100 项与 SGT-003 相关的药物交易

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