Neuromuscular Disease News and Research

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Pivotal study of olesoxime in Spinal Muscular Atrophy receives positive interim review

Pivotal study of olesoxime in Spinal Muscular Atrophy receives positive interim review

Systemic delivery of antisense oligonucleotides neutralizes mutant RNA toxicity in mice with DM1

Systemic delivery of antisense oligonucleotides neutralizes mutant RNA toxicity in mice with DM1

CUMC receives $7M to help establish Weinberg Family Cerebral Palsy Center

CUMC receives $7M to help establish Weinberg Family Cerebral Palsy Center

Pfizer, Repligen sign licensing deal to advance spinal muscular atrophy program

Pfizer, Repligen sign licensing deal to advance spinal muscular atrophy program

Leg press power best indicator of function after knee arthroplasty

Leg press power best indicator of function after knee arthroplasty

‘Gene for speed’ researcher awarded Ramaciotti Medal

‘Gene for speed’ researcher awarded Ramaciotti Medal

Two CUMC studies provide new insights into spinal muscular atrophy

Two CUMC studies provide new insights into spinal muscular atrophy

Laminin-111 protein therapy could treat Duchenne muscular dystrophy

Laminin-111 protein therapy could treat Duchenne muscular dystrophy

UI Hospital implants NeuRx Diaphragm Pacing System in a patient with ALS

UI Hospital implants NeuRx Diaphragm Pacing System in a patient with ALS

Advanced understanding of neurodegenerative diseases achieved by Trophos

Advanced understanding of neurodegenerative diseases achieved by Trophos

FDA approves Baxter's GAMMAGARD LIQUID for multifocal motor neuropathy

FDA approves Baxter's GAMMAGARD LIQUID for multifocal motor neuropathy

Diabetic Neuropathy: An interview with Dr Brian Callaghan

Diabetic Neuropathy: An interview with Dr Brian Callaghan

New understanding of how motor neurons die in spinal muscular atrophy patients

New understanding of how motor neurons die in spinal muscular atrophy patients

DYNC1H1 genetic mutation contributes to SMA-LED

DYNC1H1 genetic mutation contributes to SMA-LED

Research grants on facioscapulohumeral dystrophy prove useful as genetic basis is found

Research grants on facioscapulohumeral dystrophy prove useful as genetic basis is found

Ultragenyx reports results from UX001 Phase 1 study on HIBM

Ultragenyx reports results from UX001 Phase 1 study on HIBM

UIC researcher to explore causes of myasthenia gravis

UIC researcher to explore causes of myasthenia gravis

Positive results from Repligen's RG3039 Phase 1 study for spinal muscular atrophy

Positive results from Repligen's RG3039 Phase 1 study for spinal muscular atrophy

Genetically modified mouse mimics key features of Charcot-Marie-Tooth disease

Genetically modified mouse mimics key features of Charcot-Marie-Tooth disease

EMA grants orphan designations to Ultragenyx UX001, UX003 for treatment of HIBM and MPS 7

EMA grants orphan designations to Ultragenyx UX001, UX003 for treatment of HIBM and MPS 7