A recent Harvard study introduces a new method for treating spinal muscular atrophy. The study used "base editing" to target the disease's root cause by modifying the survival motor neuron gene. For ...
Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...
Sidra Medicine Administers Qatar's First Itvisma Gene Therapy For Spinal Muscular Atrophy. The Peninsula One-time gene ...
A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...
A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...
Itvisma should only be administered intrathecally using a lumbar puncture by health care professionals experienced in performing the procedure. The Food and Drug Administration has approved Itvisma ® ...
Two undisclosed deaths in Chinese gene-editing trials renew concerns about trial oversight, safety reporting, and transparency as gene therapy advances worldwide.
Megan covers the intersection of science and society. How is biomedical research funded? Who benefits? And how do new technologies reflect and shape our values? You can reach Megan on Signal at ...
Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Nearly six years ago, the Food and Drug ...
Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...
Spinal muscular atrophy is a rare inherited neuromuscular disorder caused by changes in the SMN1 gene. It leads to progressive muscle weakness by affecting the nerve cells that control voluntary ...
Jesy Nelson tearfully shared that the U.K. will begin universal testing for spinal muscular atrophy (SMA) — a milestone ...
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