Current understanding of and emerging treatment options for spinal muscular atrophy with respiratory distress type 1 (SMARD1)
M. Perego, Noemi Galli, Monica Nizzardo, Alessandra Govoni, Michela Taiana, Nereo Bresolin, Giacomo P. Comi, Stefania Corti
Topics & Concepts
Spinal muscular atrophySMN1MedicineSMA*Genetic enhancementDiseaseBioinformaticsRespiratory distressMotor neuronNeuroscienceIntensive care medicinePathologyPsychologyBiologyGeneSurgeryGeneticsMathematicsCombinatoricsNeurogenetic and Muscular Disorders ResearchRNA modifications and cancerRNA Research and Splicing