Results 141 to 148 of about 658 (148)

P.105INCEPTUS pre-phase 1, prospective, non-interventional, natural history run-in study to evaluate subjects aged 4 years and younger with X-linked myotubular myopathy (XLMTM)

Neuromuscular Disorders, 2019
L. Servais, P. Shieh, J. Dowling, N. Kuntz, W. Müller-Felber, B. Smith, C. Bönnemann, F. Muntoni, D. Bilder, T. Duong, R. Graham, M. Jain, M. Lawlor, V. MacBean, M. Noursalehi, T. Pitts, G. Rafferty, S. Rico, S. Prasad   +18 more
openaire   +1 more source

74PDesign of a phase 1/2 study assessing the safety and preliminary efficacy of ASP2957 gene therapy for X-linked myotubular myopathy (XLMTM)

Neuromuscular Disorders
A. Ganguli, R. Gentyala, R. Graham, O. Mayer, A. Chaparro, R. Shell, P. Li, J. Coats, F. Muntoni, K. Skjolaas, M. Lawlor, N. Kuntz, A. Dhawan, J. Dowling, L. James, H. Tran   +15 more
openaire   +1 more source

Systemic Gene Replacement Therapy for Treatment of X-Linked Myotubular Myopathy (XLMTM)

2023
exaly  

X-linked benign form of myotubular myopathy (XLMTM) with necklace fibres: A case report

2013
Ortez, C, Nascimento, A, Jou, C, Jimenez-Mallebrera, C, Paco, S, Corbera, J, Viancalana, V, Colomer, J   +7 more
openaire   +1 more source

Systemic Gene Replacement Therapy for Treatment of X-Linked MyoTubular Myopathy (XLMTM)

2016
Alan H. Beggs, Martin K. Childers
exaly  

Systemic Gene Replacement Therapy for Treatment of X-Linked Myotubular Myopathy (XLMTM)

2015
Alan H. Beggs, Martin K. Childers
exaly  

Systemic Gene Replacement Therapy for Treatment of X-Linked Myotubular Myopathy (XLMTM)

2018
exaly  

Systemic Gene Replacement Therapy for Treatment of X-Linked Myotubular Myopathy (XLMTM)

exaly