Neuromuscular Disease News and Research

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Study demonstrates therapeutic potential of new class of synthetic oligonucleotides for DMD treatment

Study demonstrates therapeutic potential of new class of synthetic oligonucleotides for DMD treatment

Isis announces top-line results from ISIS-PTP1B Rx Phase 2 study in type 2 diabetes patients

Isis announces top-line results from ISIS-PTP1B Rx Phase 2 study in type 2 diabetes patients

AMRI awarded NIH contract for drug development services

AMRI awarded NIH contract for drug development services

Therapy Box releases app that allows people with limited vocal ability to use own voices

Therapy Box releases app that allows people with limited vocal ability to use own voices

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Wimpy antibody prevents more serious self-inflicted forms of kidney disease, researchers say

Wimpy antibody prevents more serious self-inflicted forms of kidney disease, researchers say

Study: Vitamin D deficiency more prevalent in neuromuscular conditions

Study: Vitamin D deficiency more prevalent in neuromuscular conditions

Protein once seen as promising anti-cancer compound helps to stabilize neural circuits

Protein once seen as promising anti-cancer compound helps to stabilize neural circuits

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

Positive results from pivotal clinical trial of Trophos’ olesoxime in SMA patients to be presented at AAN 2014

Positive results from pivotal clinical trial of Trophos’ olesoxime in SMA patients to be presented at AAN 2014

Trophos' olesoxime shows beneficial effect on maintaining motor function in SMA patients

Trophos' olesoxime shows beneficial effect on maintaining motor function in SMA patients

Scientists receive new awards from CIRM to advance revolutionary stem cell science in medicine

Scientists receive new awards from CIRM to advance revolutionary stem cell science in medicine

ALS TDI forms research partnership with Neurimmune to advance potential treatments for ALS

ALS TDI forms research partnership with Neurimmune to advance potential treatments for ALS

Imaginative play with medically themed toys may improve recovery in chronically ill children

Imaginative play with medically themed toys may improve recovery in chronically ill children

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy