News

Researchers from Saudi Arabia demonstrated that a neurological panel assay that they developed, offers a high diagnostic yield for limb girdle muscular dystrophy (LGMD) and other myopathies.

Idera Pharmaceuticals recently presented preclinical data regarding the gene-silencing mechanisms of its third generation antisense (3GA) technology platform, a potential therapeutic for diseases that include, but are not limited to, facioscapulohumeral muscular dystrophy (FSHD). The presentations took place at the recent 12th Annual Meeting of the Oligonucleotide Therapeutics Society in Montreal,…

Summit Therapeutics announced that it has received a Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for its lead drug candidate, ezutromid, as a treatment for Duchenne muscular dystrophy (DMD). The FDA defines Rare Pediatric Diseases as disorders that primarily affect people from birth to age…

Capricor Therapeutics, Inc. announced that the U.S. Department of Defense (DoD) has awarded the company a $2.4 million grant to establish a scalable, commercially-ready process to manufacture CAP-2003, the company’s proprietary exosome product candidate. Capricor is a clinical-stage biotechnology company working on the discovery, development and commercialization of biological therapies.

Researchers at Columbia University Medical Center (CUMC) have succeeded in using high-resolution electron microscopy visualize the intracellular channel involved in the contraction of the skeletal muscle. The study, “Structural Basis for Gating and Activation of RyR1,” was published in Cell. A number of conditions are known to affect various body…

Children with Duchenne muscular dystrophy (DMD) show signs of excessive activity in certain leg muscles, an apparent reaction by the body to compensate for the instability caused by muscle weakness associated with this disease. This compensation mechanism may have a negative impact on the muscles and increase the effort necessary…

Researchers have found that a specific protein, CD82, is a marker for muscle stem cells and its expression is reduced in muscular dystrophy patients, suggesting a potential role for this previously unknown protein in the disease. The study, “CD82 Is a Marker for Prospective Isolation of Human Muscle Satellite Cells…

Monday’s announcement by the Food and Drug Administration (FDA) of accelerated approval for Exondys 51 (eteplirsen) injection, the first drug given approval for the treatment of Duchenne muscular dystrophy (DMD), was a much-celebrated likely conclusion to a debate that saw the resignation of Sarepta Therapeutics’ (makers of Exondys 51)…

In a recent article titled “Therapeutic Strategy for Heart Failure in Becker Muscular Dystrophy” and published in the International Heart Journal, Dr. Koichi Kimura of the University of Tokyo and colleagues present a review of the current strategies and suggest treatment options to treat heart failure in BMD.

The U.S. Food and Drug Administration has granted accelerated approval to Exondys 51 (eteplirsen), by Sarepta Therapeutics, making it the first drug approved to treat Duchenne muscular dystrophy (DMD) patients. Specifically, this treatment is for DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.