News

Rapamycin administered to a mouse model of dystroglycanopathy, a particular form of muscular dystrophy, worked to reduce fibrosis and inflammation, researchers reported, while enhancing muscle strength. The study, “Four-week rapamycin treatment improves muscular dystrophy in a fukutin-deficient mouse model of dystroglycanopathy,” was published in the journal Skeletal Muscle. Eystrophin-glycoprotein…

A study describing the cognitive decline over time in adults with myotonic dystrophy type 1 (DM1) was published in the scientific journal Neuromuscular Disorders. The study, titled “Cognitive decline over time in adults with myotonic dystrophy type 1: A 9-year longitudinal study” assessed the cognitive ability of the participants over a period of nine years and is the longest study to date with the largest group of participants.

Researchers identified a group of cells called fibrocytes that may contribute to inflammation and fibrosis leading to muscle loss in Duchenne muscular dystrophy (DMD). The study, “Identification And Function Of Fibrocytes In Skeletal Muscle Injury Repair And Muscular Dystrophy,” was published in The Journal of Immunology. The muscle…

Detection of the protein dysferlin may help doctors diagnose cases of Miyoshi myopathy (MM), a muscular disorder that primarily affects muscles of the hands and feet, according to a case study published in the journal Experimental And Therapeutic Medicine. In the study, “Atypical Miyoshi Distal Myopathy: A Case…

About 1 in 4 women whose sons have Duchenne or Becker muscular dystrophy were unaware of their carrier status and their own risk of cardiac problems, according to the results of a survey conducted by researchers at the University of Michigan. The study based on the survey, ”Knowledge of carrier status…

The Muscular Dystrophy Association (MDA) and Jiffy Lube service centers have been partners in the annual Muscle Up! campaign since 2012, joining forces to help people with muscular dystrophy (MD) live longer and grow stronger. On its 5th anniversary, the organizations are announcing that more than $5…

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.