ACE-083 is currently being studied in a Phase 2 trial for FSHD and another for Charcot-Marie-Tooth (CMT) disease.
Eteplirsen is the first drug approved in the US for the treatment of DMD.
The DMD space is becoming increasingly crowded as the FDA works to identify a drug worth approving.
This is the second extension requested by the FDA.
The CCDG and CMG programs aim to discover the genes and genomic variants that cause or contribute to various diseases.
Translarna (ataluren) is for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
Researchers recommend that follow-up protocols be developed for those with elevated CK but negative DMD gene testing.
The committee cited a lack of consistent clinical trial results as well as concerns over serious adverse events.
Researchers have linked the Jagged1 gene to a much milder form of muscular dystrophy in golden retrievers.
The FDA is set to make a decision on the NDA by late February, 2016.
Overall, whole exome sequencing achieved a 45% success rate in difficult-to-diagnose cases of LGMD.
A deflazacort regimen appeared to slow loss of ambulation, but not without several side effects.
The procedure may have benefits for those with stroke, Bell's palsy, and muscular dystrophy.
The guidelines emphasize a multidisciplinary approach to the diagnosis and treatment of CMDs.
The conditions are similar, but differ in rate of progression and age of onset.
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