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

Roche presents data from the risdiplam pivotal FIREFISH & SUNFISH studies in SMA Typ I-III at the 2019 AAN

Firefish Study in SMA Typ I. Babies started treatment at nearly seven months old 11 (64.7%) infants were able to sit (with or without support) while 9 (52.9%) achieved upright head control after 12 months of treatment as assessed by the Hammersmith Infant Neurological Examination Module 2 (HINE-2). Finally, 1 infant (5.9%) achieved the milestone … Fortsätt läsa Roche presents data from the risdiplam pivotal FIREFISH & SUNFISH studies in SMA Typ I-III at the 2019 AAN