You have full access to this article via your institution. Three different approaches have now been approved for the treatment of SMA: overexpression of SMN with an adeno-associated virus (AAV) vector ...

(A) Immunofluorescence images of spinal cord sections from wild-type Δ7SMA mice at 25 weeks that received AAV9-ABE + AAV9-GFP in a 10:1 ratio by neonatal ICV injection, stained for GFP to indicate AAV ...

The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy. Scientists ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

David R. Liu, PhD, a professor at Harvard University, the Broad Institute of MIT and Harvard, and the Howard Hughes Medical Institute A research team led by gene editing pioneer David R. Liu, PhD, ...

Gene therapy GC101 safely improved or stabilized motor function in children and adolescents with SMA types 2 and 3, a small ...

Spinal muscular atrophy (SMA) is a leading cause of infant mortality. Most cases of the disease are caused by mutations in a gene called SMN1 that reduce the production of functional SMN protein, ...

Zolgensma is the only drug that promises a cure for Spinal Muscular Atrophy, provided it is administered to affected children before the symptoms appear.

AMES, Iowa – Researchers at Iowa State University have discovered what could lead to a new treatment for spinal muscular atrophy, a potentially fatal genetic disorder in young children. Ravindra Singh ...

SMA type 3 is a hereditary disease that affects motor neurons. People may also refer to it as Kugelberg-Welander disease. Symptoms usually begin after 18 months of age. Children with SMA type 3 can ...

AveXis, a Novartis company, announced the US Food and Drug Administration (FDA) has approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of paediatric patients less than 2 years of age ...