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.
UC San Diego researchers launch clinical trial to test gene therapy for Alzheimer’s disease

UC San Diego researchers launch clinical trial to test gene therapy for Alzheimer’s disease

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

Canadian Neuromuscular Disease Registry increases patient access to research, clinical trials

Canadian Neuromuscular Disease Registry increases patient access to research, clinical trials

Designer DNA agent reduces myeloma stem cell abundance, lowers disease burden in mice

Designer DNA agent reduces myeloma stem cell abundance, lowers disease burden in mice

Replacing the RNA strand with DNA may enhance efficacy of ASO-based drugs

Replacing the RNA strand with DNA may enhance efficacy of ASO-based drugs

Amilorides reduce SARS-CoV-2 viral replication in vitro

Amilorides reduce SARS-CoV-2 viral replication in vitro

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