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SpinalMuscularAtrophy news

Roche drug hits the mark in spinal muscular atrophy studies

PMLiVE - Oct 7, 2018
Roche has positive clinical data on its risdiplam candidate in spinal muscular atrophy (SMA), suggesting it could add to the emerging treatment options for the devastating genetic disease. Risdiplam – which Roche licenses from PTC Therapeutics – is a ...

Roche and Novartis confirm dual threat to Biogen's biggest growth driver

Vantage - Oct 4, 2018
Competitors are advancing with potentially more effective and more convenient projects than the groups' antisense spinal muscular atrophy therapy Spinraza, Biogen's most important sales growth driver. A gene therapy from Novartis is yielding stellar ...

AveXis Files for FDA Approval of Gene Therapy to treat Spinal Muscular Atrophy Type I

Digital Journal - Oct 18, 2018
Spinal muscular atrophy (SMA) is a neuromuscular disease caused by a mutation in the gene survival motor neuron 1. In a healthy person, this gene produces a protein that is critical to the function of the nerves that control muscles. Without it, those ...

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Spinal Muscular Atrophy videos

Spinal muscular atrophy - causes, symptoms, diagnosis, treatment, pathology

What is spinal muscular atrophy (SMA)? SMA's an autosomal recessive disorder that causes alpha motor neurons in the spinal cord to die, resulting in muscle ...

Siblings with Spinal Muscular Atrophy (High School Lunch with a Disability)

Noelle is a high school student who is diagnosed with spinal muscular atrophy. Mikey, her older brother, has the same diagnosis.As Noelle faces the difficulties ...

Growing Up With Spinal Muscular Atrophy

Noël Ratapu was born with a condition that means she is gradually losing muscle strength and mobility. Now 15, Noël has found her voice and identity in the ...

A first-ever therapy for spinal muscular atrophy

Nervous and kind of excited,” 14-year-old Malorie Fox prepares to receive a new treatment.

Families of Spinal Muscular Atrophy Canada

A short look at the lives of families affected by SMA - kids participating in outdoor fun - and expressing hope for their futures. An HD version is also available for ...