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Feb 3, 2025 · Mouth opening unusually small in youngest SMA patients, study finds. Bulbar muscles, those of the face and throat, are affected in spinal muscular atrophy (SMA), and …
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How new SMA treatments are turning hope into reality
4 days ago · Last month, the biotechnology company Scholar Rock submitted an application to the U.S. Food and Drug Administration requesting approval of an experimental add-on therapy …
Cure SMA seeks $10M in DOD funding in advocacy …
4 days ago · SMA is a genetic disorder characterized by progressive muscle weakness and wasting.Over the last decades, three new SMA treatments have been approved in the U.S., and all of them have been proven to slow the …
Scholar Rock asks FDA to approve apitegromab for SMA
Jan 31, 2025 · “With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA …
Liver issues precede SMA neuromuscular symptoms: Mouse study
1 day ago · In particular, fatty liver disease — a condition marked by the buildup of fat in the liver, causing inflammation and damage — may be a common complication of SMA.Underscoring …
Spinal muscular atrophy overview - SMA News Today
Jan 24, 2025 · The most common types of SMA are caused by mutations in the SMN1 gene, leading to a deficiency of a protein called SMN, or survival motor neuron protein.. SMA is estimated to affect about 1 in every 10,000 live births …
Rectal balloon training a needed tool for coccyx mobilization
Jan 28, 2025 · Coccyx, or tailbone, mobilization, internal and external, has been a helpful treatment for one of my patients, reducing tailbone pain.For Michael, who has spinal muscular …
US, EU regulators to review higher-dose Spinraza for use in SMA
Jan 24, 2025 · Spinraza, the first disease-modifying therapy approved for SMA, is given via intrathecal injection, meaning directly into the spinal canal. The first dose is usually given as …
Levodopa therapy may ease SMA symptoms, per new …
Jan 29, 2025 · Use of levodopa therapy tested in SMA mouse model. In SMA, muscle weakness due to the shortage of a protein called SMN can cause problems with posture, and maintaining posture is important for sitting and …
High adherence to Evrysdi linked to lower patient healthcare costs
Jan 17, 2025 · Investigating impact of Evrysdi adherence on healthcare costs in real world. SMA is caused primarily by mutations in the SMN1 gene that lead to a deficiency of the survival …