Spinal Muscular Atrophy News and Research

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Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of death among infants and toddlers. Characterized by selective loss of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Over time, patients afflicted by SMA continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe. It is estimated that approximately 1 in 6,000 to 1 in 10,000 infants are born annually worldwide with SMA.
Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

ISIS conducts phase 2 study of ISIS-SMNRx in infants with type I spinal muscular atrophy

ISIS conducts phase 2 study of ISIS-SMNRx in infants with type I spinal muscular atrophy

Final patient completion of phase II study of TR040303 in patients treated for acute myocardial infarction announced by Trophos

Final patient completion of phase II study of TR040303 in patients treated for acute myocardial infarction announced by Trophos

Stem cell lines are ideal research tools for designing models to understand disease progression

Stem cell lines are ideal research tools for designing models to understand disease progression

Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

One-time treatment slows onset and progression of ALS, study finds

One-time treatment slows onset and progression of ALS, study finds

Neurodyn acquires all assets related to new drug candidate Memogain from Galantos Pharma

Neurodyn acquires all assets related to new drug candidate Memogain from Galantos Pharma

Medical investigators discover new aspect of gene-splicing process that produces mRNA

Medical investigators discover new aspect of gene-splicing process that produces mRNA

Research: RG3039 drug can extend survival, improve function in spinal muscular atrophy mouse models

Research: RG3039 drug can extend survival, improve function in spinal muscular atrophy mouse models

CGCF backs Canadian scientist's research on Spinal Muscular Atrophy

CGCF backs Canadian scientist's research on Spinal Muscular Atrophy

Isis Pharmaceuticals starts ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals starts ISIS-SMNRx Phase 2 study in infants with SMA

Clinical trial signals new era in treatment of neurodegererative disorders

Clinical trial signals new era in treatment of neurodegererative disorders

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

Researchers identify new mechanism in onset of motor neuron diseases

Researchers identify new mechanism in onset of motor neuron diseases

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

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