Boys with Duchenne muscular dystrophy (DMD) treated with the experimental gene therapy SGT-001 in the IGNITE DMD clinical trial continue to show improvements in motor and lung function after two years, according to new data presented at this year’s Muscular Dystrophy Association (MDA) Annual Meeting. “What we see…

Treatment with SGT-001 — Solid Biosciences’ gene therapy candidate for Duchenne muscular dystrophy (DMD) — improves lung function, according to data from the first six patients enrolled in the ongoing IGNITE DMD clinical trial. The improvements, seen one year after a single infusion of the SGT-001 gene therapy into…

Two more participants have been dosed in the IGNITE DMD clinical trial, bringing to eight the total number of Duchenne muscular dystrophy (DMD) patients thus far given Solid Biosciences‘ investigational gene therapy SGT-001. The U.S. trial is testing SGT-001’s safety and efficacy in boys with DMD ages…

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy SGT-001 continues to lead to improvements in physical functioning, disease-related biomarkers, and patient-reported outcomes…

Ultragenyx Pharmaceutical and Solid Biosciences announced a strategic collaboration to develop and commercialize new gene therapies for Duchenne muscular dystrophy (DMD). The collaboration will combine Solid’s gene therapy construct for DMD and Ultragenyx’s cell…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, Solid Biosciences’ investigational gene therapy for people with Duchenne muscular dystrophy (DMD), the company announced. Updated safety and effectiveness data for all patients dosed to date,…

The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial testing Solid Biosciences’ gene therapy SGT-001 for people with Duchenne muscular dystrophy (DMD), the company announced. In a letter to Solid, the FDA has requested more information about the gene therapy’s…

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK have joined forces to offer up to $1 million in funding for gene therapy research in Duchenne muscular dystrophy (DMD). The organizations are specifically looking for proposals that address the immune system’s response to gene therapy, a major challenge…

The U.S. Food and Drug Administration (FDA) is requesting more information on the process used to manufacture the gene therapy SGT-001, as it reviews the clinical hold placed on the Phase 1/2 trial of this Duchenne muscular dystrophy (DMD) therapy candidate, Solid Biosciences reported. IGNITE DMD (NCT03368742) will remain…

Solid Biosciences announced it will reduce its workforce by approximately one third to cut expenses and focus on advancing the development of SGT-001, its gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD). SGT-001 uses a viral vector to deliver a…