Results 131 to 140 of about 658 (148)

COMPOSITIONS FOR TREATING XLMTM

2023
FIELDEN MARK, TABEBORDBAR MOHAMMADSHARIF, SESHADRI SAURAV   +2 more
openaire   +1 more source

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

Neuromuscular Disorders, 2022
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   +17 more
openaire   +1 more source

A multicenter, retrospective medical record review of patients with X-Linked Myotubular myopathy (XLMTM): the RECENSUS study

Neuromuscular Disorders, 2017
A. Beggs, B. Byrne, S. de Chastonay, T. Haselkorn, I. Hughes, E. James, N. Kuntz, J. Simon, L. Swanson, M. Yang, Z. Yu, S. Yum, S. Prasad   +12 more
openaire   +1 more source

Intracranial Hemorrhage Secondary to Vitamin K Deficiency in X-Linked Myotubular Myopathy

Neuromuscular Disorders, 2021
Sabrina W Yum, Alicia M Alcamo, Oscar H Mayer   +2 more
exaly  

ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment (1053)

Neurology, 2020
Perry B. Shieh, Nancy Kuntz, Barbara Smith, James Dowling, Wolfgang Müller-Felber, Carsten G. Bönnemann, Laurent Servais, Francesco Muntoni, Astrid Blaschek, Sarah Neuhaus, Lindsay N. Alfano, Alan H. Beggs, Ana Buj-Bello, Martin (Casey) Childers, Tina Duong, Robert J. Graham, Minal Jain, Emma S. James, Michael W. Lawlor, Jun Lee, Victoria MacBean, Fulvio Mavilio, Michael Murtagh, Mojtaba Noursalehi, Suyash Prasad, Salvador Rico   +25 more
openaire   +1 more source

Neuromuscular Ultrasound for Diaphragm Assessment Following Gene Replacement Therapy in a Canine Model of X-Linked Myotubular Myopathy (XLMTM) (P05.083)

Neurology, 2013
Aarti Sarwal, J. D. Serfas, Erin Mitchell, Michael Cartwright, Anna Buj-Bello, Alan Beggs, Martin Childers   +6 more
openaire   +1 more source

Mutation Spectrum of the MTM1 Gene in XLMTM Patients: 10 Years of Experience in Prenatal and Postnatal Diagnosis

Clinical Genetics, 2016
Silvia Russo, Federica Sangiuolo, Giuseppe Novelli   +2 more
exaly  

Real-World Impact of X-Linked Myotubular Myopathy (XLMTM) on Caregivers in the United Kingdom (UK), Germany, and Spain

Value in Health
Haselkorn, T., Hughes, W., Schara-Schmidt, Ulrike, Lennox, A., Roca, A., Miller, B., Jensen, I., Solomon, F., Sanchez-de la Rosa, R., Montaner Picart, J., Braun, F., Chatwin, M., Nascimento, A.   +12 more
openaire   +2 more sources

O.39ASPIRO phase 1/2 gene therapy trial in X-linked motubular myopathy (XLMTM): update on preliminary safety and efficacy findings

Neuromuscular Disorders, 2019
J. Dowling, P. Shieh, N. Kuntz, C. Bonnemann, W. Muller-Felber, M. Lawlor, L. Servais, B. Smith, M. Noursalehi, S. Rico, S. Prasad   +10 more
openaire   +1 more source

Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)

2014
CHILDERS MARTIN K, BEGGS ALAN H, BUJ BELLO ANA MARIA   +2 more
openaire   +9 more sources