Ivanhoe on MSN11mon
SMA: A Little Boy’s Journey of Survival
SMA is caused by a defect in the survivor motor neuron one gene ... one is called SMN 1 – that’s what’s missing in spinal muscular atrophy.” Now, the first FDA-approved oral medication ...
Nature1y
The survival motor neurons protein uses its ZPR for nuclear localization
Nature Cell Biology - Spinal muscular atrophy is caused by mutations in the survival motor neurons gene, SMN1. The SMN protein localizes to both the cytoplasm and the nuclear bodies. One hallmark ...
TheHealthSite11d
Raising Awareness On Spinal Muscular Atrophy: The Silent Battle Every Indian Should Know
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standardmedia.co.ke2d
Battle against the body's own nerves: How Motor Neurone Disease alters lives
Spinal muscular atrophy (SMA) It refers to a group of ... form is caused by a mutated or missing gene known as the survival motor neuron gene 1 (SMN1), which causes the neurons to deteriorate ...
Hosted on MSN16d
Broader population could benefit from Novartis spinal muscular atrophy treatment
Analytics company GlobalData has shared its optimism regarding the future of Novartis’ spinal muscular atrophy ... biallelic mutations in the survival motor neuron 1 (SMN1) gene.
Nature1mon
Progressive muscular atrophy in the ALS spectrum
A large, retrospective analysis of patients with amyotrophic lateral sclerosis (ALS) and progressive muscular atrophy ... upper motor neuron signs in the PMA patients was unrelated to survival ...
Managed Healthcare Executive6d
FDA and EMA Accepts Applications for New Nusinersen Regimen
An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...
SMA3d
Spinal cord gray matter may be imaging marker of SMA types 2, 3
More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...
Monthly Prescribing Reference1mon
Gene Therapy Improves Motor Function in Spinal Muscular Atrophy Type 2
in patients aged 2 to less than 18 years with spinal muscular atrophy (SMA) type 2. The randomized, double-blind, sham-controlled phase 3 STEER study (ClinicalTrials.gov Identifier: NCT05089656 ...
SMA12d
High adherence to Evrysdi linked to lower patient healthcare costs
Nonadherence by SMA patients to their prescribed regimen of Evrysdi is linked to higher healthcare costs, a real-world U.S.
University of Sheffield4y
Genetic therapies for motor neuron disorders
SMA is a devastating motor neuron disease which affects children. It is caused by an abnormal survival motor neuron (SMN) gene, which leads to a reduction of SMN protein levels. Approximately 50 ...
FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
We are pleased to announce that our applications for the higher dose regimen of nusinersen are now under review in the US and Europe,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular ...