Cugamycin, a small molecule compound targeting the disease-causing RNA repeats in people with myotonic dystrophy type 1 (DM1), showed promise in improving muscle defects — without evident side effects — in an early study using mouse and cell models, researchers report.
Sarepta Therapeutics‘ casimersen (SRP-4045), one of the company’s investigational exon-skipping therapies for Duchenne muscular dystrophy (DMD), showed promising results in an interim analysis of an ongoing Phase 3 clinical trial. These positive data are expected to support the submission of a new drug application (NDA) with the…
Welcome to my first column at Muscular Dystrophy News Today. Let me tell you a little about myself. I turned 60 in January and have been dealing with muscle loss symptoms since I was 47. When I first noticed the symptoms, my form of muscular dystrophy had not…
New nine-month data on four boys with Duchenne muscular dystrophy (DMD) enrolled in Study-101 testing Sarepta Therapeutics‘ micro-dystrophin gene therapy continues to show “very encouraging” results, company officials said. These updated data, given in a recent investors’ webinar, show the gene therapy resulted in 81.2% increase in dystrophin protein…
Initial misdiagnosis is a common experience of those living with chronic illnesses. Many diseases have similar characteristics, and during the process of elimination, patients can receive an incorrect diagnosis. In previous columns, I have written about my early developmental delays. I’ve shared my tips for teachers…
My mom used to shake me awake in the morning, cook me breakfast, and send me off to school with a packed lunch. She made sure I took all my pills, ate vegetables, and had consistent physical therapy. My dad and some teachers acted as my arms and legs to…
Patients with Duchenne muscular dystrophy (DMD) and their caregivers now have a new tool to use to find an array of goods, programs, and services that can assist them with a variety of needs. The resource finder is part of the Duchenne Family Assistance Program (DFAP), an effort the…
For more than four decades, comedian Jerry Lewis hosted the MDA Labor Day Telethon on behalf of the Muscular Dystrophy Association. That annual event helped the MDA become the nation’s largest non-government source of funding for neuromuscular disease research, with more than $1.4 billion disbursed since its establishment 68…
Santhera Pharmaceuticals intends to file for a conditional marketing authorization with the European Medicines Agency (EMA) for idebenone — to be marketed as Puldysa — as a treatment for respiratory dysfunction in Duchenne muscular dystrophy (DMD). As it continues to gather data on the therapy, the company…
Cure SMA and Parent Project Muscular Dystrophy (PPMD) have formed a strategic partnership with Prometheus Research to improve data collection and analysis for patients with spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Both U.S. nonprofit groups have developed notable patient registry initiatives in the past decade. The…
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