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 ...
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 ...
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, ...
Peter L. Salgo, MD: Let’s talk about clinical sense right now. Let’s go over something like the diagnostic work-up for SMA [spinal muscular atrophy], all right? What does that include? The challenge ...
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 ...
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