Hope for a Therapeutic Drug to Treat MD

Dr. Kathryn Wagner was one of the speakers who attended the FSH Society International Patient and Researcher Meeting in Las Vegas.  (Which there will be more information on to come) She believes that ACE-031, made by Acceleron Pharma Inc., will be effective in FSHD.

ACE-031 is a drug being developed to increase muscle mass and strength to treat neuromuscular diseases.

From the Accelron website:
ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting molecules that bind to and signal through a cell surface receptor called Activin Receptor Type IIB (ActRIIB). ACE-031 is a completely human, recombinant fusion protein that is produced by joining a portion of the human ActRIIB receptor to a portion of a human antibody. This creates a freely circulating, decoy version of ActRIIB which removes proteins, such as GDF-8 (myostatin) and other related molecules that limit the growth and strength of muscle.

Dr. Wagner brought to light how little attention FSH is receiving.  Opening her talk with the statement that FSH and ALS have the same prevalence in the population.  The difference between the two is that at this time there are only 3 studies and no drug trials that  are going on for FSH, but there are 64 studies being done on ALS...... 21 of which are with novel drugs (not FDA approved).

Acceleron's press release:
Acceleron Pharma Receives FDA Fast Track Designation for ACE-031 for the Treatment of Duchenne Muscular Dystrophy

A start in the right direction.


Thanks to the FSH Society for the great speakers.

With Amanda at the FSH Society International Patient and Researcher Meeting