Cambridge, Massachusetts-based Acceleron Pharma, Inc., is now recruiting adults with FSH muscular dystrophy for a Phase 2 clinical trial of ACE-083. The initial trial site is located in St. Louis, Missouri. Additional sites across the U.S. are expected to begin recruiting soon.
ACE‐083 is an investigational drug that inhibits selected proteins in the transforming growth factor‐beta (TGF‐β) superfamily involved in the regulation of muscle size and strength. ACE‐083 has been designed to increase muscle size and strength specifically in the muscles into which the drug is administered. Acceleron is developing ACE‐083 for diseases in which improved muscle strength in a specific set of muscles may provide a clinical benefit to patients, such as FSHD. As an “investigational” agent, ACE‐083 is not approved by any regulatory agency for use in any country.
For full details of the clinical trial, including updates on new clinical trial locations, please visit ClinicalTrials.gov: Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD).
Additional details can be downloaded here:
ACE-083
Would love to participate and as a minimum be kept up to date.
I have this condition muscle weakness in the tibialis anterior In both my lower legs. More so in the left than right. All the doctors I have seen in my area say there is nothing they can do except where braces. I am 71 and extremely active. Cycling, alpine skiing and resistance training and I even use Electronic muscle stimulation. I used to walk a lot too but with the foot drop in both legs it is very uncomfortable to do. With all that I cannot get muscles to grow. My left leg thigh is approximately 25% smaller in size than my right. Both calf muscles have deteriorated significantly in size. After testing the first neuro doctor said I had CMT others after that said no it was just a pinched nerve in my lower back. Can anyone help me?
Please consult a neuromuscular specialist who is well-trained in all of the muscular dystrophies. Your symptoms are consistent with FSH muscular dystrophy, and that can be confirmed through a genetic test. You might also consult other orthotists to see whether there might be a more comfortable brace for you. Some of our members have been very happy with the newer carbon fiber designs.