Exondys 51 (eteplirsen) was approved by the U.S. FDA for the treatment of Duchenne muscular dystrophy (DMD) on Sept. 19, but the drug helps only 13 percent of those battling the disease. Now, CureDuchenne is redoubling its efforts to find treatments for the 87 percent of DMD…
At Catabasis Pharmaceuticals’ first Investor Day earlier this month, the company provided an in-depth review of its strategy and development pipeline of product candidates for treating rare diseases, including the Duchenne muscular dystrophy drug edasalonexent (CAT-1004). Guest speakers included Craig McDonald, MD, director of the NeuroNEXT Program at…
Researchers recently created a 3D image of the structure and composition of the skeletal muscle, including muscle affected by fibrosis, using multiple imaging techniques coupled with quantitative stereology. The study from a research team at UC San Diego and the Rehabilitation Institute of Chicago, titled “High-resolution three-dimensional reconstruction of fibrotic skeletal muscle…
This video from Muscular Dystrophy WA shares Laura’s story. Laura is a 22-year-old university student from Western Australia who has Emery-Dreifuss muscular dystrophy. She’s studying for a Bachelor of Criminology and Justice with the hope of one day working with criminal offenders on parole. Find out more…
Exercising and keeping fit is always encouraged, but when it comes to muscular dystrophy (MD), there are many patients who cannot partake in physical exercise. For others, engaging in an exercise routine can help improve muscle tone and increase overall fitness and health. However, you should always consult your doctor…
The American Society of Gene and Cell Therapy (ASGCT), the primary professional body for gene and cell therapy, recently produced a guide on therapeutic gene editing to inform policy-makers, patient advocates, and the general public about the technique.
A retrospective analysis of the brain structure by MRI shows alterations in white matter in patients with myotonic dystrophy type 1 (DM1) disease. These findings suggest the brain is involved in DM1 in addition to the muscular and systemic symptoms already affecting these patients. The study, “MRI findings and cognitive…
https://www.youtube.com/watch?v=wsASIV_fiMo In this video from Michelle Durante, we see demonstrations of exercises designed to help muscular dystrophy patients. Michelle talks about some of the types of muscular dystrophy and how adapted exercises can help slow the progression of the disease. Find out more about the nine different types…
Summit Therapeutics has enrolled its first U.S. patients in its proof-of-concept Phase 2 clinical trial of ezutromid in patients with Duchenne muscular dystrophy (DMD). The study is actively recruiting patients. “Ezutromid has shown great promise in preclinical testing as a universal treatment that has the potential to slow…
Duchenne and Becker are two of the most common types of muscular dystrophy (MD). Both types of the disease mainly affect boys, with Duchenne MD (DMD)Â usually being diagnosed between the ages of three and five, and Becker MD (BMD) between the ages of 11 and 25. Although they tend to…
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