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Spinal Muscular Atrophy

Delivery Method for SMA Drug Nusinersen Evaluated for Safety and Efficacy

A new, more streamlined method of administration has recently been developed for the use of nusinersen in Spinal muscular atrophy (SMA).

Spinal Muscular Atrophy

NICE Recommends Drug Treatment for SMA

NICE issued a draft guidance recommending the use of risdiplam as a new drug treatment for spinal muscular atrophy (SMA)

Spinal Muscular Atrophy

Pilot Program on Newborn Screening to Find & Treat SMA

A pilot program in Australia looked at at the importance of testing for spinal muscular atrophy (SMA) in newborns.

Spinal Muscular Atrophy

Gene Therapy Restores Muscle Function in Kids with Spinal Muscular Atrophy

Spinal muscular atrophy is a debilitating rare illness, but advances in gene therapy might prove to be the future of treatment.

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