FDA clears Phase 2 study of exon skipping therapy for DMD
2023年4月18日 · The results showed that dystrophin protein levels increased to a mean of 10.27% of normal in patients treated with the low dose, and to 15.79% of normal in those …
Muscular Dystrophy News Home | Muscular Dystrophy News
4 天之前 · MDA honors researcher, patient advocate with 2025 Legacy Awards. The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two …
Sevasemten lowers markers of muscle damage in BMD: Trial
2024年12月17日 · “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of …
Duchenne muscular dystrophy: Exercise and physical therapy
2024年6月17日 · A physical therapist knowledgeable about DMD and progressive muscle weakness and other DMD symptoms can create a therapeutic exercise program that safely …
Educating all ages about living with muscular dystrophy
2024年10月7日 · In my four decades of life with limb-girdle muscular dystrophy, I’ve become comfortable educating everyone I meet about my quality of life and the ways all of us living …
Sarepta stops development of exon 51-skipping therapy for DMD
2024年11月12日 · SRP-5051 was a next-generation successor to Exondys 51 (eteplirsen), an older exon 51-skipping therapy also from Sarepta. Exondys 51 is conditionally approved in the …
Your DMD community: Resources and support - Muscular …
2023年7月13日 · While Duchenne muscular dystrophy (DMD) is one of the most common types of this group of muscle-wasting disorders, affecting about 1 in 3,500 male births, each patient’s …
Trial will test gene therapy for limb-girdle muscular dystrophy type
2024年3月28日 · Phase 1b study of ATA-200 to enroll 6 children with LGMD-2C/R5. Mutations in the SGCG gene are thought to prevent the sarcoglycan complex from stabilizing dystrophin — …
Rare pediatric disease designation granted to LAMA2-CMD therapy
2024年10月3日 · The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that Modalis Therapeutics is …
MDA Engage Symposium: Stanford, CA | Muscular Dystrophy News
This Engage Symposium will provide updates on all neuromuscular diseases including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), amyotrophic lateral sclerosis …