News

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…

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…

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…

Researchers have found that certain cognitive functions, specifically those requiring sequential processing of auditory and visual information, do not improve with age in patients with Duchenne muscular dystrophy (DMD). The study, “Profile of cognitive function in adults with duchenne muscular dystrophy,” was published in the journal Brain and…

Researchers from Australia recently reviewed experimental and clinical data from multiple research projects and clinical trials in an attempt to determine whether nutraceutical supplements could benefit or prolong the health of Duchenne MD patients. The findings suggest that some of these alternative products could be a valuable complementary therapy for…

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) favors the renewal of the conditional marketing authorization of PTC Therapeutics’ Translarna (ataluren), an investigational drug in development for ambulatory patients 5 years and older, who have nonsense mutation Duchenne muscular dystrophy…

Women who received their initial risk assessment of being carriers of Duchenne muscular dystrophy (DMD) before 2003 should be reassessed in order to provide them with the right counseling about the risks of having a child with DMD and developing cardiomyopathy, according to a study published in the scientific journal Neurology Genetics.