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MuscularAtrophy news

AveXis Flies, But PTC Sinks On Spinal Muscular Atrophy Data

Investor's Business Daily - Oct 3, 2017
Spinal muscular atrophy robs patients of physical strength by affecting nerve cells in the spinal cord. The Phase 1 trial was designed to evaluate the safety and efficiency of AVXS-101 in infants, known as Type 1 patients. AveXis looked to extend the ...

Pivotal Study for SMA Treatment Begins, Triggers $20M Milestone Payment

Rare Disease Report - Oct 12, 2017
... between PTC Therapeutics, Inc., Roche and the SMA Foundation (SMAF) has transitioned into the pivotal part of the study that will evaluate the safety and efficacy of RG7916 in pediatric and adult patients with Type 2/3 spinal muscular atrophy (SMA).

Feasibility of combined treatment for type III spinal muscular atrophy: a pilot study

Dove Medical Press - Sep 27, 2017
Objective: To find solutions and improve methods of therapy for patients suffering from type III spinal muscular atrophy (SMA-III) by way of a combination of conventional treatment (medicines, physiotherapy) and fetal stem cells (FSCs) transplantation ...

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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 ...

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.

Growing up with Spinal Muscular Atrophy

Maija Fredrichberg has Spinal Muscular Atrophy. Her muscles are slowly deteriorating and her parents were told she wouldn't live past her fifth birthday. Maija is ...

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 ...