News

A Phase 1/2 trial evaluating Sarepta’s microdystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD) was placed on clinical hold by the U.S. Food and Drug Administration. The FDA found trace amounts of plasmid DNA…

Insilico Medicine and A2A Pharmaceuticals, two leading artificial intelligence (AI) biotechs, are collaborating to create a new company called Consortium.AI, which will advance AI for the development of new molecules to treat Duchenne muscular dystrophy (DMD) and other orphan diseases. Consortium.AI will feature computational new candidates designed…

Either by coincidence or by design, MDA Hellas, the Muscular Dystrophy Association of Greece, is located on Elpidos Street in downtown Athens. Elpidos is Greek for “hope” — and that’s exactly what MDA Hellas offers the many neuromuscular disease patients under its care. Antigone Karras is executive manager of…

In the mythology of ancient Greece, Hermes was the god of boundaries and transitions, with the ability to move between the mortal and divine worlds. In modern-day Athens, Hermes Christos Athanasiou Gill is a cheerful, trilingual 7-year-old who likes math and enjoys playing Minecraft, a video game. He also has…

Treatment of Duchenne muscular dystrophy (DMD) patients with Raxone (idebenone) delays the need for assisted ventilation by three years, new data from a Phase 3 clinical trial show. Trial results also demonstrate that measuring the air flow speed in and out of the lungs, or peak expiratory flow…

Benitec Biopharma recently granted Axovant Sciences worldwide rights to its investigational AXO-AAV-OPMD gene therapy program for the treatment of oculopharyngeal muscular dystrophy (OPMD) under a global licensing agreement. AXO-AAV-OPMD is currently in preclinical development, and Axovant plans to launch a placebo-controlled trial testing the therapy candidate for OPMD…

Coping with Duchenne muscular dystrophy is difficult enough in the United States. But in neighboring Mexico, people with Duchenne often face social discrimination, lack of access to the newest therapies, a financially strapped healthcare system, and general ignorance. “Very few Mexicans know anything about this disease,” said Graciela Méndez Covarrubias,…

Acceleron’s ACE-083, for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA), the company announced. FSDH affects about 20,000 people in the U.S. It is a rare genetic muscle disorder for which there…

Five years after the death of a Utah boy named Mitchell Jones, the national foundation created in his name helps families struggling with the ravages of Duchenne muscular dystrophy. Mitchell, who was diagnosed with Duchenne at the age of 3, died of heart failure on March 2, 2013, just…

A global Phase 3 trial evaluating the efficacy and safety of oral edasalonexent in patients with Duchenne muscular dystrophy (DMD) will begin enrolling boys, ages 4 to 7, in the coming months, Catabasis Pharmaceuticals announced. Top-line results from this trial, to be called POLARIS, are expected in April 2020, and this…