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.
Newborn screening for spinal muscular atrophy promises a benefit

Newborn screening for spinal muscular atrophy promises a benefit

New form of gene editing slows ALS progression in mice

New form of gene editing slows ALS progression in mice

New breakthrough offers hope for potential treatment of rare childhood epilepsy

New breakthrough offers hope for potential treatment of rare childhood epilepsy

Outcomes-based drug reimbursement may be on the horizon, report says

Outcomes-based drug reimbursement may be on the horizon, report says

Successful gene therapy trial in patients with X-linked Chronic Septic Granulomatosis

Successful gene therapy trial in patients with X-linked Chronic Septic Granulomatosis

Study identifies molecular 'bridge' between ALS-causing genes

Study identifies molecular 'bridge' between ALS-causing genes

Dog study suggests that AAV in gene therapy could induce cancer

Dog study suggests that AAV in gene therapy could induce cancer

Neuroscientists develop new method for uniquely targeted gene therapy

Neuroscientists develop new method for uniquely targeted gene therapy

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

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