Biomarkers of therapeutic efficacy in adolescents and adults with 5q spinal muscular atrophy: a systematic review
Maria Gavriilaki, Vasileios Papaliagkas, Alexandra Stamperna, Maria Moschou, Konstantinos Notas, Sotirios Papagiannopoulos, Marianthi Arnaoutoglou, Vasilios Κ. Kimiskidis
Topics & Concepts
MedicineSpinal muscular atrophyBiomarkerSMA*PopulationInternal medicineClinical trialSurrogate endpointRandomized controlled trialOncologyPhysical medicine and rehabilitationBioinformaticsPathologyPhysical therapyDiseaseEnvironmental healthChemistryBiochemistryMathematicsCombinatoricsBiologyNeurogenetic and Muscular Disorders ResearchSpinal Dysraphism and MalformationsCardiomyopathy and Myosin Studies