Cure SMA 2019

Roche at Cure SMA 2019

Presentations

Jun 30, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy

CureSMA FIREFISH Motor Function Presentation 2019: FIREFISH Part 1: 1-year results on motor function in infants with Type 1 spinal muscular atrophy (SMA) receiving risdiplam (RG7916)

Jun 30, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy

CureSMA FIREFISH Survival Presentation 2019: FIREFISH Part 1: Survival, ventilation and swallowing ability in infants with Type 1 spinal muscular atrophy (SMA) receiving risdiplam (RG7916)

Jun 30, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy

CureSMA SUNFISH Presentation 2019: Update from SUNFISH Part 1: Safety, tolerability and PK/PD from the dose-finding study, including exploratory efficacy data, in patients with type 2 or 3 spinal muscular atrophy (SMA) treated with risdiplam (RG7916)

Posters

Jun 28, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy

CureSMA JEWELFISH Poster 2019: JEWELFISH: Risdiplam (RG7916) increased survival of motor neuron (SMN) protein levels in patients with spinal muscular atrophy (SMA) that have previously received therapies targeting SMN2 splicing, or olesoxime

Jun 28, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy

CureSMA FIREFISH Modified Bayley Poster 2019: Adaptation of the gross motor subscale of the Bayley Scales of Infant and Toddler Development, third edition (BSID-III) simplifies assessment for infants with Type 1 symptomatic spinal muscular atrophy (SMA)

Jun 28, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy

CureSMA MFM 32 Poster 2019: Validity and reliability of the Motor Function Measure (MFM32) in children with neuromuscular disorders, and in individuals with Type 2 and non-ambulant Type 3 spinal muscular atrophy (SMA)