A new therapeutic paradigm has heightened the need for meaningful measures of assessing patients. As new treatments have become available for people with 5q spinal muscular atrophy (SMA), a new report ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented 1-year data from the pivotal Part 2 of SUNFISH, a global ...
The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric scales for adults and the need for modified evaluations to accurately measure ...
Increased motor function as measured by Revised Upper Limb Module (RULM)** and the Hammersmith Functional Motor Scale-Expanded (HFMSE)*** between months 12 and 24. Stabilized motor function for ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Patients treated with onasemnogene abeparvovec recorded ...
The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.
Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the ...
Evrysdi, a survival motor neuron 2-directed RNA splicing modifier, both sustained and improved motor function at 24 months in children and adults with type 2 or type 3 spinal muscular atrophy, ...
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