The newly approved therapy Vyondys 53 (golodirsen) is available for immediate distribution to treat people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, according to the medicine’s developer Sarepta Therapeutics. ... Read more

Treatment with gene therapy candidate SGT-001 may lead to clinically meaningful improvements for patients with Duchenne muscular dystrophy (DMD), new data from two participants in the Phase 1/2 IGNITE ... Read more

The world of employment and disabilities is difficult for me to understand. Readers of my column have always been good about giving feedback. I’d love your input on this ... Read more

The experimental therapy ATL1102 is safe and continues to show evidence of improved upper muscle strength and function in all nine non-ambulatory boys with Duchenne muscular dystrophy (DMD) in ... Read more

Finding the perfect Christmas gift requires listening intently to the recipient’s wants and needs. This year, the social media team at Muscular Dystrophy News Today asked followers what would ... Read more

Following its failure to increase dystrophin levels of patients in an early-stage clinical trial, Wave Life Sciences has discontinued development of the investigational exon-skipping therapy suvodirsen for people with ... Read more

I realize that having a full-time job as well as Duchenne muscular dystrophy can be challenging. Balancing my time between self-care, including stretching and doctor’s appointments, and work can ... Read more

The U.S. Food and Drug Administration (FDA) has authorized use of the first test intended to facilitate newborn screening for Duchenne muscular dystrophy (DMD). “Diagnostics that can safely and ... Read more

The U.S. Food and Drug Administration has conditionally approved Vyondys 53 (golodirsen) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. This accelerated approval comes less than ... Read more

GPM Investments, the sixth largest convenience store chain in the U.S., is running its “Tis the Season for Giving” holiday campaign throughout December to support the Muscular Dystrophy Association ... Read more