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Spinal Muscular Atrophy: Monthly Roundup

by Nisha Cooch | Jun 3, 2019

Much of the most recent research regarding spinal muscular atrophy (SMA) has focused on understanding the mechanisms mediating the disease as well as the clinical value of different pharmaceutical interventions. There have been several reviews on the latter topic,...

Spinal Muscular Atrophy: Monthly Roundup

by Nisha Cooch | Jun 3, 2019

Much of the most recent research regarding spinal muscular atrophy (SMA) has focused on understanding the mechanisms mediating the disease as well as the clinical value of different pharmaceutical interventions. There have been several reviews on the latter topic,...

FDA Approves AveXis’ Gene Therapy Zolgensma to Treat SMA in Children Up to Age 2

by Michael Nace | May 24, 2019

Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now an approved and potential “one-time” intravenous treatment for pre-symptomatic newborns through 2-year-olds with any type of SMA, the U.S. Food and Drug...

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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Patient and Parent Perspectives on the Use of Nusinersen for Spinal Muscular Atrophy

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 Muscular Atrophy

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

How Salbutamol May Help Patients with Spinal Muscular Atrophy: New Insights

The vast majority of spinal muscular atrophy (SMA) cases are caused by a mutation in the survival motor neuron 1 (SMN1) gene, which leads ...Read more

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Potential Relationship Found Between Early Non-Neuromuscular Symptoms and the Lack of SMN Protein in SMA Patients

A direct relationship may exist between early non-neuromuscular symptoms and the lack of survival motor neuron (SMN) protein directly ...Read more
Scientists Create New Method to Calculate Number of SMN1 Copies and Identify SMA Carriers

Scientists have created a new method to calculate the number of SMN1 gene copies present in individuals and identify possible ...Read more
New Endurance Tests Can Measure Fatigue in SMA Patients Types 2-4, Study Reports

Endurance tests that capture upper and lower body fatigue in spinal muscular atrophy patients — across ages, from children to adults, ...Read more

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Spinraza Dose #7

I go in for my seventh Spinraza injection tomorrow. Like I’ve said before, it’s just kind of become another part of my routine ...Read more
Risdiplam Shows Potential in Babies and Adults with SMA

Risdiplam, an investigational oral therapy, shows higher SMN protein levels in both pediatric and adult SMA patients, according to an ...Read more
Preparing for Injection Days

In Michael Morale’s personal YouTube channel smajourney51, he did a video this week about preparing for injection days. He does ...Read more
Strategies for Combatting Migraines and Fatigue

I used to get migraines all the time, but now I get them much less frequently. Still, with the recent storm and dramatic shifts in the ...Read more

Spinal Muscular Atrophy: Monthly Roundup

Spinal Muscular Atrophy: Monthly Roundup

FDA Approves AveXis’ Gene Therapy Zolgensma to Treat SMA in Children Up to Age 2

Reldesemtiv Improves Muscle Strength in SMA Patients, Trial Data Show

Latest Posts from SMA News Today

Recent SMA Neurologist News

Neurology Related Forum Topics

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