학술논문
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
Document Type
Article
Author
Lawlor, M.W.; Beatka, M.; Prom, M.J.; Ott, E.; Danielson, S.; Krakau, P.; Kumar, S.N.; Meng, H.; Vanden Avond, M.; Wells, C.; Schoser, B.; Margeta, M.; Sewry, C.A.; Jones, K.A.; Shieh, P.B.; Qasim, U.R.; Kuntz, N.L.; Smith, B.K.; Dowling, J.J.; Tsuchiya, E.; Müller-Felber, W.; Blaschek, A.; Bönnemann, C.G.; Neuhaus, S.; Foley, A.R.; Saade, D.N.; Seferian, A.M.; Gordish-Dressman, H.; Beggs, A.H.; Christensen, S.; Conner, E.; James, E.S.; Lee, J.; Sadhu, C.; Miller, W.; Sepulveda, B.; Varfaj, F.; Prasad, S.; Rico, S.
Source
In: eBioMedicine . (eBioMedicine, January 2024, 99)
Subject
Language
English
ISSN
23523964