Throughout 2019, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other important events related to muscular dystrophy. As we look forward to bringing you more news this year, we present the 10 most-read stories of 2019. No. 10 – “Sarepta…
I sometimes wonder how I will survive Chicago winters going forward. Winter is not a problem for some people with neuromuscular disorders. But for those of us in colder climates, it’s a big deal. The reality of my city’s wet and slippery conditions means I need to live creatively…
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. In an email reply to Muscular Dystrophy News Today, a spokeswoman added that, similar to Exondys 51 (eteplirsen, also…
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 DMD trial suggest. Solid Biosciences, which developed the gene therapy, is currently working with the U.S. Food…
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 subject because it is so important. The numbers don’t make sense to me. As a society, we can do better for…
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 a nearly complete Phase 2 trial in Australia, updated data show. Final results from the open-label study, testing ATL1102…
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 be on their wish lists to make their lives easier and more comfortable. These recommendations are sure to put a…
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 Duchenne muscular dystrophy (DMD). Lack of dystrophin, an essential protein for healthy muscles, causes DMD. Patients may…
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 be physically and emotionally draining. That’s why you must find a job that is a good fit for your situation.
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 effectively screen newborns can help health care professionals identify and discuss potential treatment options with parents and caregivers before symptoms…
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