orphan drug

In a phase 1 trial in healthy volunteers, Repligen's experimental therapy for spinal muscular atrophy was safe and well-tolerated

posted on April 25, 2012 - 4:00am
Update (June 21, 2012): This story was updated to include the fact that MDA funding helped support the phase 1 trial, as well as the preclinical development of RG3039.

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