O.06 Long term outcomes for X-Linked myotubular Myopathy (XLMTM) with gene replacement therapy, resamirigene bilparvovec: Preliminary results from ASPIRO

Authors :: P. Shieh
N. Kuntz
J. Dowling
W. Müller-Felber
A. Blaschek
C. Bönnemann
R. Foley
D. Saade
A. Seferian
L. Servais
A. Bowden
M. Sarazen
J. Coats
N. Lakshman
C. Han
S. Prasad
S. Rico
W. Miller
Source :: Neuromuscular Disorders. 32:S44
Publication Year :: 2022
Publisher :: Elsevier BV, 2022.

Subjects :: Neurology
Pediatrics, Perinatology and Child Health
Neurology (clinical)
Genetics (clinical)

Tools