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)

Pivotal-SUNFISH study of Risdiplam (RG7916) continues to show significant improvement in patients with type 2/3 spinal muscular atrophy

Roche’s risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 spinal muscular atrophy Study demonstrated statistically significant improvements in the overall study population with Type 2 or 3 SMA No treatment related safety findings leading to withdrawal seen in any risdiplam trial to date Data will be shared with … Fortsätt läsa Pivotal-SUNFISH study of Risdiplam (RG7916) continues to show significant improvement in patients with type 2/3 spinal muscular atrophy

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