Clinical Experience With Gene Therapy in Older Patients With Spinal Muscular Atrophy
Susan Matesanz, Vanessa Battista, Jean Flickinger, Jennifer N. Jones, Elizabeth Kichula
Topics & Concepts
MedicineSpinal muscular atrophyPediatricsClinical trialAtrophySMA*ConfoundingSurgeryInternal medicineDiseaseMathematicsCombinatoricsNeurogenetic and Muscular Disorders ResearchHereditary Neurological DisordersCardiomyopathy and Myosin Studies