OP018: ASPIRO gene therapy trial in X-Linked Myotubular Myopathy (XLMTM): Update on preliminary efficacy and safety findings
Perry B. Shieh, Nancy L. Kuntz, James J. Dowling, Wolfgang Müller‐Felber, C. Bönnemann, Dimah Foley, A. Seferian, Laurent Servais, Michael W. Lawlor, Mojtaba Noursalehi, Suyash Prasad, Salvador Rico, Weston P. Miller
Topics & Concepts
MedicineGenetic enhancementGenePhysical therapyBioinformaticsInternal medicineGeneticsBiologyCellular transport and secretionUbiquitin and proteasome pathwaysCardiomyopathy and Myosin Studies