Two years risdiplam: New data from SUNFISH, JEWELFISH in infants, children, adults with spinal muscular atrophy (SMA)

Roche announces new 2-year risdiplam data to SUNFISH Part 1 showed risdiplam significantly improved motor function after 24 months of treatment in people aged 2-25 years with Types 2 or 3 SMA JEWELFISH study preliminary 12 month data in previously treated patients showed rapid and sustained increases in SMN protein levels Safety in SUNFISH and … Fortsätt läsa Two years risdiplam: New data from SUNFISH, JEWELFISH in infants, children, adults with spinal muscular atrophy (SMA)

New clinical trial to Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)

Roche presents its new data on the SUNFISH, JEWELFISH, RAINBOWFISH study Exploratory efficacy data showed 88% of infants treated with Evrysdi were alive and did not require permanent ventilation at two years 59% of infants were able to sit without support for at least 5 seconds No new safety signals were identified In August, the … Fortsätt läsa New clinical trial to Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)

Risdiplam-treated type 1 babies with SMA continue to achieve major motor milestones

All clinical trials involving risdiplam continue to demonstrate strong safety profile Filing for broad labels in 2019 (US) and 1H’20 (EU) PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data demonstrating the ongoing benefit of risdiplam (RG7916) for the treatment of all types of spinal muscular atrophy (SMA) at the 24th International Annual Congress of the … Fortsätt läsa Risdiplam-treated type 1 babies with SMA continue to achieve major motor milestones