Neuromuscular Disorder News and Research

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C-Path and FARA announce launch of Friedreich's Ataxia Integrated Clinical Database

C-Path and FARA announce launch of Friedreich's Ataxia Integrated Clinical Database

TGen identifies polio-like virus as potential cause of Acute Flaccid Myelitis outbreak

TGen identifies polio-like virus as potential cause of Acute Flaccid Myelitis outbreak

ALS and frontotemporal dementia share genetic connections

ALS and frontotemporal dementia share genetic connections

New medication improves motor function in children with later-onset spinal muscular atrophy

New medication improves motor function in children with later-onset spinal muscular atrophy

Newborn screening for SMA favored by most people, study finds

Newborn screening for SMA favored by most people, study finds

Single infusion of wildtype HSPCs halts cellular damage caused by neuromuscular disorder in mice

Single infusion of wildtype HSPCs halts cellular damage caused by neuromuscular disorder in mice

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

New study lays foundation for future gene replacement therapies to treat ALS patients

New study lays foundation for future gene replacement therapies to treat ALS patients

Researchers identify factors that increase risk of cardiac arrest during pediatric spine surgeries

Researchers identify factors that increase risk of cardiac arrest during pediatric spine surgeries

Study on fruit flies, brewer's yeast may provide clues about cause of Spinal Muscular Atrophy

Study on fruit flies, brewer's yeast may provide clues about cause of Spinal Muscular Atrophy

Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

Scientists reveal mechanism underlying cellular degeneration of upper motor neurons

Scientists reveal mechanism underlying cellular degeneration of upper motor neurons

2014 DIA patient fellows illustrate the power of patient advocacy

2014 DIA patient fellows illustrate the power of patient advocacy

Edison Pharmaceuticals' EPI-743 granted orphan designation for Leigh syndrome in Japan

Edison Pharmaceuticals' EPI-743 granted orphan designation for Leigh syndrome in Japan

New mouse study indicates that mutant protein in muscle cells is responsible for SBMA

New mouse study indicates that mutant protein in muscle cells is responsible for SBMA

Plant pigment could prevent damage to nerves linked with childhood form of SMA

Plant pigment could prevent damage to nerves linked with childhood form of SMA

Researchers develop valid humanized model for facioscapulohumeral muscular dystrophy

Researchers develop valid humanized model for facioscapulohumeral muscular dystrophy

Joint research program in SMA enters initial stage of clinical development

Joint research program in SMA enters initial stage of clinical development

Study focuses on vaccine-preventable illnesses in myasthenia gravis patients

Study focuses on vaccine-preventable illnesses in myasthenia gravis patients

The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies

The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies