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According to a Boston Business Journal story, Cambridge, Massachusetts-based biotech Acceleron Pharma has plans to test its ACE-083 compound in facioscapulohumeral muscular dystrophy patients in a phase 2 clinical trial later this year: “The company presented top-line data from a Phase 1 trial in healthy volunteers showing the drug, which is injected into the muscle, was able to increase the size of a patients thigh muscle by up to 14 percent versus placebo. CEO John Knopf described the results as “unprecedented” and said that the company plans to advance ACE-083 into a phase 2 trials in patients with facioscapulohumeral muscular dystrophy in mid-2016.”
This is great news. If all goes well, when do you expect treatment for all who are affected?
Hard to say. Acceleron is planning a Phase 2 trial, which could take 1-2 years depending on how fast they can recruit participants. They then need to analyze the data and if it looks promising, make a case to the FDA to allow a Phase 3 trial, which is a larger study to prove efficacy. That could take an additional 1-2 years, and then they need to apply for FDA approval.
Are they looking for participants for the study.
Not yet. We will keep our eyes open for the trial launch. You can also check clinicaltrials.gov for FSHD clinical trials.
No matter how long it takes. The mere fact that it is a priority and checked it’s a good news. By the way also we will be happy to take a part in the plan.
An increase of muscle mass doesn’t mean a gain in muscle strength…
It as been shown in trials on inhibition of myostatin if I remember well (I’m not sure…).
What’s your idea on that subject ?
Thank you in advance for your answer.
Sylvie, from AFM FSH group, France
We agree that this is the major unanswered question with regard to myostatin inhibitors in FSHD. The only way we will know is through a carefully designed clinical trial.
There is a myostatin inhibitor trial that is further ahead. Some results available below: Keep track of the trial on the ppmd website. Yes, I know that it is being tested on DMD boys but I participated in a previous webinar and was assured it would also work on FSH.
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Forgot to add this webinar is March 30, 2016 at 1 pn EST!