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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...

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...

Nerve Cell Function Impacted When Movement is Limited, Mouse Study Suggests

by Michael Nace | Jun 18, 2018

Neurological health depends as much on signals sent by the body's leg muscles to the brain as it does on those sent from the brain to the muscles, according to a recent study. These results offer new clues as to why patients with neurological diseases such as spinal...

#CureSMA2018 – Exicure’s SNA Formulation of Nusinersen Raises SMN Protein Levels with Lesser Toxicity, Mouse Study Reports

by Michael Nace | Jun 15, 2018

Exicure’s gene c prolongs survival, increases the amount of SMN proteins, and shows limited toxicity in a mouse model of the disease, study data show. The investigational compound is a spherical nucleic acid (SNA) formulation of nusinersen, the active substance in...

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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

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

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

Nerve Cell Function Impacted When Movement is Limited, Mouse Study Suggests

#CureSMA2018 – Exicure’s SNA Formulation of Nusinersen Raises SMN Protein Levels with Lesser Toxicity, Mouse Study Reports

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