Muscular Atrophy News and Research

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Researchers find novel ways to improve participation in clinical research

Researchers find novel ways to improve participation in clinical research

Scientists discover how potential new drug targets spinal muscular atrophy

Scientists discover how potential new drug targets spinal muscular atrophy

Therapeutic RNA corrects error in genetic processing that leads to familial dysautonomia

Therapeutic RNA corrects error in genetic processing that leads to familial dysautonomia

Investigational drug may help increase protein levels in babies with spinal muscular atrophy

Investigational drug may help increase protein levels in babies with spinal muscular atrophy

Scientists discover basic molecular mechanism that helps understand how ALS works

Scientists discover basic molecular mechanism that helps understand how ALS works

Impaired transport processes in neurons contribute to neurodegenerative disorders

Impaired transport processes in neurons contribute to neurodegenerative disorders

Researchers develop method to identify splicing-based biomarkers for liver cancer

Researchers develop method to identify splicing-based biomarkers for liver cancer

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’