Frontiers3d
Clinical Phenotyping and Biomarkers in Spinal and Bulbar Muscular Atrophy
Background: Spinal and bulbar muscular atrophy (SBMA) or Kennedy disease [OMIM: 313200] is a rare X-linked neuromuscular disease. Patients commonly present with muscle cramps, tremors, leg weakness, ...
KYUK10h
Spinal stimulation restored muscles wasted by rare genetic disorder
Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal ...
Healio3d
FDA receives biologics license application for apitegromab in spinal muscular atrophy
A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...
News Medical on MSN1d
Neurostimulation improves movement in adults with spinal muscle atrophy
A new drug-free, minimally invasive intervention targets the root cause of progressive loss of neural function in spinal muscle atrophy (SMA), an inherited neuromuscular disease.
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Infant with HDFN and spinal muscular atrophy successfully treated
A case report described an infant with concurrent HDFN and SMA who was succesfully treated with an onasemnogene abeparvovec ...