So in type 1, which mainly affects the infants or the little babies, and so those little babies are usually symptomatic or present with weakness fairly early on in their lifetime within the first ...

The parents of two-year-old Aakriti Singh diagnosed with Spinal Muscular Atrophy – SMA type-1, are worried about her next ...

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More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...

The mutation that causes Spinal Muscular Atrophy (SMA) can be detected in a ... For example, children with a moderate phenotype (type II SMA) typically need mobility assistance at some point ...

For children with SMA, assistive devices -- walkers, wheelchairs, standers, and more -- are the key to staying active. Learn which ones may help your child.

A man with a muscle-wasting disease is pleased Manitoba is asking a federal agency to reconsider its opposition to a ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

Topline results of a phase 3 clinical trial of an investigational gene therapy for spinal muscular atrophy type 2 found the treatment ... on data from the phase 1/2 open-label STRONG clinical ...