A recent review provides insight into the clinical progression of treatment-naive patients with spinal muscular atrophy type 3 who have lost ambulation. Patients with spinal muscular atrophy (SMA) ...
"The availability of disease-modifying therapies for patients with other types of SMA suggests that these key disease characteristics in SMA 3 patients should be amenable to therapy." — Barry J. Byrne ...
The new report is based on real-world data from a patient registry and shows some patients even regain motor function under treatment with nusinersen for spinal muscular atrophy (SMA). A new ...
In two clinical trials, Evrysdi improved motor function in people living with SMA over a broad spectrum of ages and levels of disease severity, including Types 1, 2, and 3 SMA Evrysdi helped infants ...
man MS wheelchair Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the ...
Please provide your email address to receive an email when new articles are posted on . Biogen announced it intends to initiate a global phase 3b study to investigate clinical outcomes and safety of a ...
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, ...
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window BOSTON -- Older ...
Scholar Rock's stock price has increased from $6 to $16, indicating increased market interest. Scholar Rock develops apitegromab, a targeted therapy for Spinal Muscular Atrophy (SMA) that inhibits ...
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