News

Santhera Pharmaceuticals, a Swiss company focused on the treatment of mitochondrial and neuromuscular diseases, recently announced that it has discussed with the U.S. Food and Drug Administration (FDA) its plans of submitting a New Drug Application (NDA) for Raxone ® /Catena ® (idebenone), intended as a therapy for…

A new study recently published in the International Journal of Clinical and Experimental Pathology characterized the immune factors involved in the pathogenesis of dysferlinopathy in patients. The study is entitled “CD4(+) cells, macrophages, MHC-I and C5b-9 involve the pathogenesis of dysferlinopathy” and was conducted by researchers…

The U.S. Food and Drug Administration (FDA) granted orphan drug status to an investigational therapy being developed by MediciNova, Inc. to treat Krabbe disease. The company expects MN-166 (ibudilast) to become a treatment option for the rare genetic degenerative condition that affects children at early age, causing the weakening of…

The Muscular Dystrophy Association is offering an exclusive opportunity to those who want to participate in the sold out 2015 Bank of America Chicago and Marine Corps Marathons. The MDA is offering a secure, guaranteed entry by signing up through MDA Team Momentum, the organization’s nationwide endurance program. Space, however,…

Merozyne Therapeutics AB recently initiated a project with the goal of developing a pharmaceutical treatment to address laminin alpha2 chain-deficient congenital muscular dystrophy (MDC1A) through supporting the research that Madeleine Durbeej-Hjalt and Virginie Carmignac conducted at Lund University. Merozyne Therapeutics AB has been established very recently by several entrepreneurial life science firms based at the Medicon…

The 5th Annual Getzlaf Golf Shootout to benefit CureDuchenne will take place on September 12th at Sutra Lounge in Costa Mesa, California and September 13th at the Monarch Beach Golf Links in Dana Point, California. The event will be hosted by the captain of the Anaheim Ducks, …

In a new study entitled “Under-recognition of Low Blood Pressure Readings in Patients with Duchenne Muscular Dystrophy,” researchers determined for the first time how blood pressure is affected in patients with duchenne muscular dystrophy. They report a significant portion of DMD patients exhibit low blood pressure, and highlight the need for clinicians…

The FSH Society, a Massachusetts-based charity supporting research into facioscapulohumeral muscular dystrophy (FSHD), has announced that it recently completed the 2nd edition of the FSHD Trial Preparedness Workshop, which was held between May 29 and 30, 2015 at the University of Rochester Medical Center in Rochester, New York. The workshop included over 50 stakeholders from around…

A new study published in the journal Human Molecular Genetics revealed a new therapeutic approach that could potentially alter the progression of muscle damage and dysfunction in patients with myotonic dystrophy. The study is entitled “TWEAK/Fn14, a pathway and novel therapeutic target in myotonic…

In a recent study published in Oncotarget, researchers found an inverse correlation between the level of muscle fibrosis, utrophin and the number of revertant myofibers in Duchenne Muscular Dystrophy (DMD). Results from this study reveal common links between the fibrotic and utrophin-synthesis pathways and offer new insights into…