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.
Study reveals link between alterations in RNA splicing and Alzheimer's disease

Study reveals link between alterations in RNA splicing and Alzheimer's disease

Medical professional offers unique perspective as father of a child with rare movement disorder

Medical professional offers unique perspective as father of a child with rare movement disorder

Scientists move one step closer to discovering the causes of immune diseases

Scientists move one step closer to discovering the causes of immune diseases

Faster, cheaper, better: improved gene therapy tool production

Faster, cheaper, better: improved gene therapy tool production

BUSM researchers develop advanced protocol to improve gene therapy tool production

BUSM researchers develop advanced protocol to improve gene therapy tool production

Study: Gene editing may correct genetic mutation responsible for Duchenne muscular dystrophy

Study: Gene editing may correct genetic mutation responsible for Duchenne muscular dystrophy

Statement related to data inaccuracies with newly approved gene therapy

Statement related to data inaccuracies with newly approved gene therapy

New vaccine shows promise against toughest HIV strains

New vaccine shows promise against toughest HIV strains

BIA Separations selected to support AveXis’ gene therapy pipeline

BIA Separations selected to support AveXis’ gene therapy pipeline

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

$150,000 grant will support research on spinal muscular atrophy

$150,000 grant will support research on spinal muscular atrophy

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Unique gene therapy approach paves new way to tackle rare, inherited diseases

New form of therapy may stop or reverse progressive vision loss

New form of therapy may stop or reverse progressive vision loss

Biogen and Eisai present detailed results from phase 1b study of aducanumab at CTAD meeting

Biogen and Eisai present detailed results from phase 1b study of aducanumab at CTAD meeting

Novartis: Lancet publishes pioneering study of Aimovig’s efficacy in episodic migraine patients

Novartis: Lancet publishes pioneering study of Aimovig’s efficacy in episodic migraine patients

Genentech announces clinical data from studies investigating risdiplam in spinal muscular atrophy

Genentech announces clinical data from studies investigating risdiplam in spinal muscular atrophy

Researchers discover how nerve cells control movement

Researchers discover how nerve cells control movement

Urine exRNA may be source of biomarkers for muscular dystrophy

Urine exRNA may be source of biomarkers for muscular dystrophy

Biogen and Eisai announce results of LTE Phase 1b study of aducanumab for treating MCI

Biogen and Eisai announce results of LTE Phase 1b study of aducanumab for treating MCI

Study links spinal muscular atrophy disease expression with haplotypes

Study links spinal muscular atrophy disease expression with haplotypes