News

Non-profit organization Parent Project Muscular Dystrophy (PPMD) has nominated Duke Children’s Hospital & Health Center as a Certified Duchenne Care Center, a designation that confirms the center’s quality in the treatment of Duchenne muscular dystrophy. Duke Children’s is now the ninth center in the country to be…

A novel study from Max Planck Institute for Heart and Lung Research investigators revealed a novel protein, Prmt5, as a crucial player in the regulation of muscle stem cells during muscle regeneration. The study entitled “Prmt5 is a regulator of muscle stem cell expansion in adult mice”…

This week researchers from the Institute for Neurodegenerative Disease, Massachusetts General Hospital (MGH), Harvard Medical School, released results from a study showing that a key protein associated with the development of Alzheimer’s disease, Amyloid precursor protein (APP), could potentially play an important role in eye and muscle health.  The study,…

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…

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…

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…