SMA Newsroom

ResearchNov 21, 2022

New publication: Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy.

Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study.

Outcome measures traditionally used in spinal muscular atrophy (SMA) clinical trials are inadequate to assess the full range of disease severity. The aim of this study was to assess the psychometric properties of a set of existing questionnaires and new items, gathering information on the impact of SMA from the patient and caregiver perspectives.

ResearchOct 13, 2022

New two-year data from the JEWELFISH study evaluating Evrysdi (risdiplam)

Roche announces new two-year data from the JEWELFISH study evaluating Evrysdi (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 months to 60 years at the time of enrolment. The data will be first presented at the 27th World Muscle Society (WMS) congress, 11-15 October 2022. 

It will be also announced during the Roche PAG Scientific Meeting taking place during our 3rd Scientific Congress on Spinal Muscular Atrophy in Barcelona on Saturday, 22 October at 4 pm.