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.
Complex RNA structures could have untapped therapeutic potential in the fight against COVID-19

Complex RNA structures could have untapped therapeutic potential in the fight against COVID-19

New data reveals safety, efficacy of nusinersen drug delivery method for SMA patients with advanced disease

New data reveals safety, efficacy of nusinersen drug delivery method for SMA patients with advanced disease

CHOP researchers develop tool to monitor disease severity in patients with mitochondrial myopathy

CHOP researchers develop tool to monitor disease severity in patients with mitochondrial myopathy

Innovative therapeutic platform for blood-to-brain delivery of antisense oligonucleotide therapy

Innovative therapeutic platform for blood-to-brain delivery of antisense oligonucleotide therapy

Newborn screening and early treatment for spinal muscular atrophy can save both lives and money

Newborn screening and early treatment for spinal muscular atrophy can save both lives and money

Dying patients with rare diseases struggle to get experimental therapies

Dying patients with rare diseases struggle to get experimental therapies

New components for oligonucleotide drugs show safety, efficacy in treating spinal muscular atrophy

New components for oligonucleotide drugs show safety, efficacy in treating spinal muscular atrophy

Intranasal COVID-19 therapeutic shows promise in preclinical trials

Intranasal COVID-19 therapeutic shows promise in preclinical trials

Researchers identify genetic cause of a rare neurological disorder

Researchers identify genetic cause of a rare neurological disorder

Genethon announces dosing of first patient at pediatric clinical trial platform for neuromuscular diseases

Genethon announces dosing of first patient at pediatric clinical trial platform for neuromuscular diseases

Keele researcher awarded prestigious grant to identify treatments for devastating childhood disease

Keele researcher awarded prestigious grant to identify treatments for devastating childhood disease

Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients

Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients

Study: Early benefit assessment of new drugs within the framework of AM-NOG procedure

Study: Early benefit assessment of new drugs within the framework of AM-NOG procedure

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