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.
New medication improves motor function in children with later-onset spinal muscular atrophy

New medication improves motor function in children with later-onset spinal muscular atrophy

Scientists uncover the structure of microtubule motor proteins

Scientists uncover the structure of microtubule motor proteins

UNC gene therapy scientists uncover viral barcode to penetrate blood-brain barrier

UNC gene therapy scientists uncover viral barcode to penetrate blood-brain barrier

Researchers discover gene network that regulates the motor neuron development in growing embryo

Researchers discover gene network that regulates the motor neuron development in growing embryo

Researchers describe severe toxicity with AAV9-based high-dose gene therapy in animal models

Researchers describe severe toxicity with AAV9-based high-dose gene therapy in animal models

Newborn screening for SMA favored by most people, study finds

Newborn screening for SMA favored by most people, study finds

Massachusetts grabs spotlight by proposing new twist on Medicaid drug coverage

Massachusetts grabs spotlight by proposing new twist on Medicaid drug coverage

MDA announces 13 new research grants to advance treatment for neuromuscular diseases

MDA announces 13 new research grants to advance treatment for neuromuscular diseases

Drug can safely and effectively help infants with SMA gain muscle function, study shows

Drug can safely and effectively help infants with SMA gain muscle function, study shows

Biogen and Ionis awarded 2017 Prix Galien USA Award for SPINRAZA

Biogen and Ionis awarded 2017 Prix Galien USA Award for SPINRAZA

Scientists directly convert skin cells from healthy adults into motor neurons

Scientists directly convert skin cells from healthy adults into motor neurons

New drug shows promising results for treating spinal muscular atrophy

New drug shows promising results for treating spinal muscular atrophy

Drug puts a $750,000 ‘price tag on life’

Drug puts a $750,000 ‘price tag on life’

Study shows how RNA splicing errors may trigger development of ALS, FTD

Study shows how RNA splicing errors may trigger development of ALS, FTD

Study unravels new clues to cause and treatment of spinal muscular atrophy

Study unravels new clues to cause and treatment of spinal muscular atrophy

New gene variant linked to Lou Gehrig's disease

New gene variant linked to Lou Gehrig's disease

Researchers uncover new potential target for treating spinal muscular atrophy

Researchers uncover new potential target for treating spinal muscular atrophy

Positive CHMP opinion recognizes efficacy of Biogen’s SPINRAZA for treatment of spinal muscular atrophy

Positive CHMP opinion recognizes efficacy of Biogen’s SPINRAZA for treatment of spinal muscular atrophy

Targeted treatment slows progression of two degenerative brain diseases in mice

Targeted treatment slows progression of two degenerative brain diseases in mice

Researchers use mathematical modeling to raise density of bones badly degraded by osteoporosis

Researchers use mathematical modeling to raise density of bones badly degraded by osteoporosis