Muscular Atrophy News and Research

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Splicing factor enhances ability of triple negative breast cancers to grow and metastasize

Splicing factor enhances ability of triple negative breast cancers to grow and metastasize

Adult SMA patients treated with Nusinersen show improvement in motor function

Adult SMA patients treated with Nusinersen show improvement in motor function

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

Study finds attractive therapeutic target for treatment of Kennedy's disease and prostrate cancer

Study finds attractive therapeutic target for treatment of Kennedy's disease and prostrate cancer

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

Quanterix’ Simoa technology powers more than 85 percent of Neurofilament light biomarker research to be unveiled at American Academy of Neurology Annual Meeting

Quanterix’ Simoa technology powers more than 85 percent of Neurofilament light biomarker research to be unveiled at American Academy of Neurology Annual Meeting

$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