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.
Investigating the prerequisites for developing RNA-based antibiotics

Investigating the prerequisites for developing RNA-based antibiotics

Early identification and treatment can reduce financial costs associated with spinal muscular atrophy

Early identification and treatment can reduce financial costs associated with spinal muscular atrophy

New fund could enable early access to life-saving treatments for NHS patients in England

New fund could enable early access to life-saving treatments for NHS patients in England

Plastins may promote disease when they act like workaholics and disrupt cellular environment

Plastins may promote disease when they act like workaholics and disrupt cellular environment

UW Medicine scientists contribute to the publication of first complete, gapless genome sequence

UW Medicine scientists contribute to the publication of first complete, gapless genome sequence

Researchers find a way to interfere with the energy pathway that promotes liver cancer spread

Researchers find a way to interfere with the energy pathway that promotes liver cancer spread

New approach using RNA therapeutic can fix protein problems in cystic fibrosis

New approach using RNA therapeutic can fix protein problems in cystic fibrosis

JPND announces call for multinational research on understanding the mechanisms of non-pharmacological interventions

JPND announces call for multinational research on understanding the mechanisms of non-pharmacological interventions

Single-patient pilot study shows the ability of antisense oligonucleotide to suppress mutant ALS gene

Single-patient pilot study shows the ability of antisense oligonucleotide to suppress mutant ALS gene

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