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Reldesemtiv Improves Muscle Strength in SMA Patients, Trial Data Show

by Michael Nace | Aug 14, 2018

Reldesemtiv improved muscle strength for walking and respiration in a dose-dependent manner in spinal muscular atrophy (SMA) patients, data from a Phase 2 clinical trial show. As a fast muscle activator, reldesemtiv will not treat the underlying causes of SMA — a lack...

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by poindexter | Jul 9, 2018

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With Spinraza Available, Cure SMA Chief Urges Nationwide Screening for Disease

by Michael Nace | Jul 9, 2018

Over the next 20 years, the number of Americans with spinal muscular atrophy (SMA) will rise from 10,000 to nearly 15,000 — and 8,000 of those living with SMA today will still be alive come 2038, predicts Kenneth Hobby, president of Cure SMA. The Chicago-based...

New Way of Restoring SMN Protein Levels by Targeting RNA Holds Promise, Study Says

by Michael Nace | Jun 25, 2018

A new way of restoring potentially adequate levels of survival motor neuron (SMN) — the protein missing in spinal muscular atrophy (SMA) — uses small molecules that target the structure of an intermediate player in this protein's production. The study on this...

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Patient and Parent Perspectives on the Use of...

At the end of 2016, the U.S. Food and Drug Administration (FDA) approved nusinersen for the treatment of spinal muscular atrophy (SMA).1 ...Read more

Motor Unit Number Index as a Biomarker for Spinal...

Major challenges in the clinical care of those with spinal muscular atrophy (SMA) are determining patients’ clinical stages, ...Read more

Eosinophilic Oesophagitis in Spinal Muscular...

Though rare, spinal muscular atrophy (SMA) does sometimes co-occur with other rare conditions. Both SMA type 2 and eosinophilic ...Read more

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New Gene Therapy Approach Able to Repair Mutations Causing SMA, Other Inherited Diseases, Mouse Study Suggests

A new gene therapy approach using RNA molecules called transfer RNAs (tRNAs) was able to repair a subset of mutations causing spinal ...Read more
Rural Pennsylvania Clinic Treats Amish, Mennonite Children Who Have SMA

Visitors to the Clinic for Special Children (CSC) just outside Strasburg, Pennsylvania, might be forgiven for thinking they’ve ...Read more
New Online Patient Education Program on Gene and Cell Therapies Launched

The American Society of Gene and Cell Therapy (ASGCT) has unveiled a new online resource designed to keep patients, caregivers, and the ...Read more
Scientists Re-create All Human SMA Types, Including Type 4, in Fruit Fly Models

Researchers from the University of North Carolina have described 14 different fruit fly models that mimic all the types of spinal ...Read more

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