Spinal Muscular Atrophy News and Research

RSS
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 study explores how human plastins behave in cells

New study explores how human plastins behave in cells

Study identifies new therapy to treat cystic fibrosis

Study identifies new therapy to treat cystic fibrosis

Mutations in GEMIN5 protein can cause rare genetic disorders in children

Mutations in GEMIN5 protein can cause rare genetic disorders in children

First-in-human clinical trial to test safety, efficacy of gene therapy for Alzheimer's disease

First-in-human clinical trial to test safety, efficacy of gene therapy for Alzheimer's disease

Investigational antisense medicine lowers disease burden, reduces myeloma cell abundance in mice

Investigational antisense medicine lowers disease burden, reduces myeloma cell abundance in mice

New approach can prevent toxicity linked to neurological gene therapy

New approach can prevent toxicity linked to neurological gene therapy

Study reveals new gene involved in motor neuron diseases

Study reveals new gene involved in motor neuron diseases

Study looks into safety and early outcomes of gene therapy for spinal muscular atrophy

Study looks into safety and early outcomes of gene therapy for spinal muscular atrophy

Method can help detect specific glial cells integral to synapses

Method can help detect specific glial cells integral to synapses