Duchenne Muscular Dystrophy
DMD is predominately caused by out-of-frame deletions in the dystrophin gene, which results in absent or defective dystrophin protein, leading to progressive and irreversible muscle function loss.
Investigators aim to facilitate early recognition and consideration for patients with Duchenne muscular dystrophy and internalizing psychiatric symptoms.
SGT-001 is a novel adeno-associated viral vector mediated gene transfer that could potentially address the underlying genetic cause of DMD, mutations in the dystrophin gene.
Patients who received deflazacort were older when they reached disease milestones compared with patients taking prednisolone or prednisone.
Emflaza is approved for the treatment of Duchenne muscular dystrophy regardless of genetic etiology.
Patients who received ACE inhibitors showed a slower progression of myocardial fibrosis and fewer cardiovascular events.
Eteplirsen is the first drug approved in the US for the treatment of DMD.
Over 52 weeks, patients taking deflazacort experienced improved muscle strength and less weight gain compared with prednisone.
Deflazacort is intended to help improve muscle strength and other functional outcomes in DMD regardless of genetic etiology.
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 panel took a similar stance in the fall when it did not recommend Biomarin's drisapersen for the treatment of DMD.
After a lukewarm review, the FDA has officially denied the NDA for drisapersen.
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.
A deflazacort regimen appeared to slow loss of ambulation, but not without several side effects.
The conditions are similar, but differ in rate of progression and age of onset.
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