Research Briefs: ALS, DMD, MTM and SMA

Duchenne muscular dystrophy

Acceleron Pharma announced Aug. 4 that it has received fast track designation from the U.S. Food and Drug Administration (FDA) for its experimental compound ACE031 for the treatment of Duchenne muscular dystrophy (DMD). ACE031 is designed to interfere with the actions of myostatin, a protein that inhibits muscle growth.

BioMarin Pharmaceutical announced Aug. 2 that it will not pursue development of BMN195, an experimental compound designed to increase production of a protein called utrophin, for the treatment of DMD. Utrophin is believed to at least partially substitute for the dystrophin protein, which is missing in DMD-affected muscles. A trial in healthy volunteers showed the drug did not reach adequate blood concentrations to achieve its intended effect.

Researchers in the Netherlands are studying the effects of physical training in boys with DMD to see whether such training can slow down the portion of muscle deterioration that's due to "disuse" in this disease. See No Use is Disuse study posting.

Spinal muscular atrophy

Repligen Corp. is developing RG-SMA, an experimental drug in a class called DcpS inhibitors. It's designed to increase production of the SMN protein, a deficiency of which is the underlying cause of spinal muscular atrophy (SMA).

Amyotrophic lateral sclerosis and SMA

The French biotech company Trophos is testing its experimental compound TRO19622, also known as olesoxime, in patients with amyotrophic lateral sclerosis (ALS) in Europe and is planning a clinical trial in spinal muscular atrophy (SMA). The drug is designed to promote the function and survival of nerve cells damaged in these diseases by interacting with cellular mitochondria. The ALS trial is no longer recruiting participants.

Myotubular myopathy

A research group supported in part by MDA has identified Labrador retrievers that naturally develop a disease that mimics humanX-linked myotubular myopathy clinically and genetically. The finding should help veterinarians diagnose the condition in dogs and is likely to be useful in studying the human disease. See MTM1 mutation associated with X-linked myotubular myopathy in Labrador retrievers

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