Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies with Risdiplam

risdiplam- RG7916 is well tolerated at all dose levels with no drug-related safety findings – SOUTH PLAINFIELD, N.J., June 16, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy […]

The Drug Development Process > Phase II clinical trials with Reldesemtiv in Patients with Spinal Muscular Atrophy (SMA II-III)

presented in an oral presentation by John W. Day, MD, Ph.D., Professor of Neurology and Paediatrics (Genetics), Stanford University. This hypothesis-generating study met its primary goal. It fulfills the effect and safety profile. In collaboration with Astellas, Cytokinetics is developing reldesemtiv as a potential treatment for people with SMA and certain other debilitating diseases and […]