Pharmacotherapy for Spinal Muscular Atrophy in Babies and Children: A Review of Approved and Experimental Therapies
Claudia A. Chiriboga
Topics & Concepts
Spinal muscular atrophySMN1SMA*MedicineGenetic enhancementMotor neuronAdeno-associated virusBioinformaticsInternal medicineGeneGeneticsBiologyVector (molecular biology)DiseaseRecombinant DNACombinatoricsMathematicsNeurogenetic and Muscular Disorders ResearchRNA modifications and cancerCongenital Anomalies and Fetal Surgery