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.
Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Researchers develop telomerase gene therapy for treating different pathologies

Researchers develop telomerase gene therapy for treating different pathologies

Study finds how the brain 're-wires' after neurological disease

Study finds how the brain 're-wires' after neurological disease

Study implicates new gene as driver of motor neuron diseases

Study implicates new gene as driver of motor neuron diseases

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

FDA approves first oral drug for treatment of spinal muscular atrophy

FDA approves first oral drug for treatment of spinal muscular atrophy

Researchers develop new therapeutic approach to treat CLN3 Batten disease

Researchers develop new therapeutic approach to treat CLN3 Batten disease

C-Path gets positive response from FDA for drug-induced skeletal muscle injury safety biomarkers

C-Path gets positive response from FDA for drug-induced skeletal muscle injury safety biomarkers

New editorial published following adverse events of gene therapy clinical trial

New editorial published following adverse events of gene therapy clinical trial

Developing Vaccines and Therapeutics by Exploiting Structure in Spherical Nucleic Acids

Developing Vaccines and Therapeutics by Exploiting Structure in Spherical Nucleic Acids

Researchers develop a potential treatment approach for Pelizaeus-Merzbacher disease

Researchers develop a potential treatment approach for Pelizaeus-Merzbacher disease

Researchers identify new molecules to study specific glial cells integral to synapses

Researchers identify new molecules to study specific glial cells integral to synapses

New therapeutic approach for Parkinson’s and other neurodegenerative diseases

New therapeutic approach for Parkinson’s and other neurodegenerative diseases

German researchers recommend early screening and treatment for spinal muscular atrophy

German researchers recommend early screening and treatment for spinal muscular atrophy

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