CURE SMA Canada > Fighting for treatments

Tick, Tock, Tick Tock the clock runs out. A day turns into a week, a week becomes a month, months turn into years – Years these people do not have. Children around the world are struggling with their families for an access to the only treatment for Spinal Muscular Atrophy, which are currently available Cure […]

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 […]