A review of advances in spinal muscular atrophy treatment agents
Objective To introduce the progress of medications used to treat spinal muscular atrophy(SMA),and to provide ideas for the research of new medications.Methods The literatures about the mechanism and treatment medications of SMA were retrieved and summarized.Results SMA is an inherited neuromuscular disease arising from homozygous loss or mutation of the survival motor neuron gene(SMN1),the main symptom is muscle weakness,medications work by altering splicing and cutting,delivering SMN1 gene copies into cells or neuroprotection.Moreover,Ca2+was found to have the potential relationship with disease phenotype.Existing researches are mainly based on the above-mentioned patterns and targets.Conclusion The comprehension of existing medications helps us to understand the mechanism of disease and the way medications work,which lays the foundation for drug development.
spinal muscular atrophypathogenesistherapeutic drug
NETL
NSTL
万方数据
维普
