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Latest Duchenne muscular dystrophy Stories

2014-02-10 20:21:08

Call on FDA for Accelerated Approval of Promising Duchenne Muscular Dystrophy Treatment WASHINGTON, Feb. 10, 2014 /PRNewswire-iReach/ -- Health staffers from over 80 Congressional offices together with agency and industry officials jammed a briefing room last Friday to hear mothers, clinicians and leading researchers call on the FDA to accept and promptly review a new drug application for eteplirsen, a promising new treatment for Duchenne Muscular Dystrophy that could save thousands...

2014-01-24 08:23:47

SOUTH PLAINFIELD, N.J., Jan. 24, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) (PTC) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the Company's marketing authorization application (MAA) for conditional approval of ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The response from the CHMP is consistent with the Company's previous...

2013-10-30 15:35:50

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a...


Word of the Day
call-note
  • The call or cry of a bird or other animal to its mate or its young.
'Call-note' is newer than 'bird-call,' which originally referred to 'an instrument for imitating the note of birds' but now also refers to 'the song or cry of a bird.'
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