Biogen said that new data examining of its already approved spinal muscular atrophy treatment Spinraza (nusinersen) indicates ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 ...

Spinal muscular atrophy (SMA) is a rare genetic disorder that causes muscle weakness and disability, typically in young children. Learn about symptoms, diagnosis, treatment options, and support.

BiogenBIIB said Wednesday its experimental treatment helped children with a devastating muscle-wasting disease even after they received an approved gene therapy from NovartisNVS.The company already ...

Living with spinal muscular atrophy. DIMITAR DILKOFF/AFP via Getty Images. By Jason Mast. March 19, 2025. General Assignment Reporter Jason Mast [email protected] ...

Source Reference: Finkel RS, et al "Risdiplam for prenatal therapy of spinal muscular atrophy" N Engl J Med 2025; DOI: 10.1056/NEJMc2300802.

Spinal muscular atrophy (SMA) causes progressive muscular atrophy, weakness, and bulbar dysfunction. It can lead to death, particularly in patients with a homozygous deletion of exon 7 or exons 7 ...

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child ...

Spinal bulbar muscular atrophy (SBMA) is a progressive genetic neuromuscular condition that affects motor neurons. Over time, it may cause symptoms such as difficulty speaking, swallowing, or walking.

Biohaven's spinal muscular atrophy drug fails to meet study goal. By Mariam E Sunny. November 25, 2024 7:06 PM UTC Updated November 25, 2024 Companies; Biohaven Ltd. Follow. Biogen ...

Scholar Rock’s stock soars more than 315% on positive trial of treatment for spinal muscular atrophy Wedbush cheered the data and said the focus now shifts to approval and commercialization.

Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal stimulation. Accessibility links. Skip to main content; ...