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  • ReveraGen BioPharma’s DMD Drug Vamorolone Gets FDA Fast Track Designation March 27, 2017
    The investigational drug vamorolone (formerly VBP15), under development by ReveraGen BioPharma, has received U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of Duchenne muscular dystrophy (DMD). This designation can speed the review of efficacy and safety data for vamorolone in boys with DMD, potentially leading to more rapid regulatory approval. Vamorolone, […]
  • FDA Gives Akashi Green Light to Resume Clinical Development of DMD Drug HT-100 March 24, 2017
    Akashi Therapeutics announced on Wednesday that the U.S. Food and Drug Administration (FDA) has cleared the way for the company to resume clinical development of HT-100 in boys with Duchenne muscular dystrophy (DMD). The news comes a year after Akashi suspended dosing and enrollment in the phase 1b/2a HALO clinical trial to test HT-100 in […]
  • Of Mice and Scientists: Joe Akmakjian Reports from the 2017 MDA Scientific Conference March 24, 2017
    I hate mice. Rodents are my worst fear. I don’t know where this incapacitating aversion comes from, but this past week I found myself faced with the undeniable truth that these abominable vermin are actually working to save my life. OK, technically researchers in teams all over the world who are conducting MDA-funded studies are […]
  • Unplugged March 23, 2017
    Cords were wrapped around my foot and tangled in my flip flop, so jumbled I could not tell where the mess began. Each cord was connected to a cell phone, a tablet or a laptop. I looked down and sighed. As I unraveled myself from the tangle I began to think about how much we […]
  • A Future Built on MDA Research: Meet Garin, the Budding Architect Shamrockstar March 22, 2017
    When asked how Duchenne muscular dystrophy (DMD) impacts her son, Garin, Rebekah Stepp puts it in terms many families can relate to. “Many parents complain that their child is difficult to wake up in the mornings. Others complain that their kids fuss over what they will wear to school. Still others complain that their kids […]


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