Tag : Muscular

Neurology

The facial nerve atrophy with spinal and bulbar muscular atrophy patients (SBMA): Three case reports with 3D fast imaging employing steady-state acquisition (FIESTA)

Newsemia
Spinal and bulbar muscular atrophy (SBMA) is an X-linked neuromuscular disease caused by a CAG repeat expansion in the androgen receptor (AR) gene [1]. Men...
Neurology

Reader response: Evidence in focus: Nusinersen use in spinal muscular atrophy: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology

Newsemia
Nusinersen is effective for patients with spinal muscular atrophy (SMA) and has been approved in the United States, Europe, and Japan.1 No life-threatening adverse events...
Neurology

Editors' note: Evidence in focus: Nusinersen use in spinal muscular atrophy: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology

Newsemia
In the article “Evidence in focus: Nusinersen use in spinal muscular atrophy,” Michelson et al. systematically reviewed 4 published clinical trials of nusinersen for patients...
Pharma / Biotech

The Pharmacokinetics of 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy.

Newsemia
Related Articles The Pharmacokinetics of 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy. Nucleic Acid Ther. 2019 Aug 20;:...

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