Roche to Share Ongoing Work on Elevidys Gene Therapy at Muscular Dystrophy Association (MDA) 2025 Conference
At the Muscular Dystrophy Association (MDA) 2025 Conference, Roche will discuss their ongoing work on Elevidys Gene Therapy.
Late-breaking oral on Elevidys’ Embark two-year data...
Dyne Therapeutics Announces Unprecedented and Sustained Functional Improvement Through 18 Months from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy
Dyne Therapeutics, a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced new long-term clinical data...
SonoThera will Present New Data of Genetic Medicine Using Targeted, Ultrasound-Mediated Delivery (UMD) in Treating Duchenne Muscular Dystrophy
SonoThera will present new data at the 2025 MDA Conference demonstrating the potential of genetic medicine using targeted, Ultrasound-Mediated Delivery (UMD) in treating Duchenne...
Solid Biosciences Plans to Request a Meeting with the FDA in mid-2025 to Discuss Potential Accelerated Approval Pathways for SGT-003
Solid Biosciences, a life sciences company developing precision, next generation, genetic medicines for neuromuscular and cardiac diseases, reported financial results for the fourth quarter...
GenAssist Announces FDA Approval of New Drug Application for GEN6050X Exon 50 Skipping Therapy
On March 06, 2025, GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in genome medicines, is thrilled to announce that it has received...