Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.

Shieh PB, Kuntz NL, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Servais L, Smith BK, Muntoni F, Blaschek A, Foley AR, Saade DN, Neuhaus S, Alfano LN, Beggs AH, Buj-Bello A, Childers MK, Duong T, Graham RJ, Jain M, Coats J, MacBean V, James ES, Lee J, Mavilio F, Miller W, Varfaj F, Murtagh M, Han C, Noursalehi M, Lawlor MW, Prasad S, Rico S

Open source

DOI: 10.1016/s1474-4422(23)00313-7
Published: 2023 Dec
Container: The Lancet. Neurology
Publisher: Not recorded
Open access: unknown

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Cite this work

BibTeX

@article{allodium:10.1016/s1474-4422-23-00313-7,
  title = {Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.},
  author = {Shieh PB and Kuntz NL and Dowling JJ and Müller-Felber W and Bönnemann CG and Seferian AM and Servais L and Smith BK and Muntoni F and Blaschek A and Foley AR and Saade DN and Neuhaus S and Alfano LN and Beggs AH and Buj-Bello A and Childers MK and Duong T and Graham RJ and Jain M and Coats J and MacBean V and James ES and Lee J and Mavilio F and Miller W and Varfaj F and Murtagh M and Han C and Noursalehi M and Lawlor MW and Prasad S and Rico S},
  year = {2023},
  journal = {The Lancet. Neurology},
  doi = {10.1016/s1474-4422(23)00313-7},
  url = {https://doi.org/10.1016/s1474-4422(23)00313-7}
}

RIS

TY  - JOUR
TI  - Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
AU  - Shieh PB
AU  - Kuntz NL
AU  - Dowling JJ
AU  - Müller-Felber W
AU  - Bönnemann CG
AU  - Seferian AM
AU  - Servais L
AU  - Smith BK
AU  - Muntoni F
AU  - Blaschek A
AU  - Foley AR
AU  - Saade DN
AU  - Neuhaus S
AU  - Alfano LN
AU  - Beggs AH
AU  - Buj-Bello A
AU  - Childers MK
AU  - Duong T
AU  - Graham RJ
AU  - Jain M
AU  - Coats J
AU  - MacBean V
AU  - James ES
AU  - Lee J
AU  - Mavilio F
AU  - Miller W
AU  - Varfaj F
AU  - Murtagh M
AU  - Han C
AU  - Noursalehi M
AU  - Lawlor MW
AU  - Prasad S
AU  - Rico S
PY  - 2023
JO  - The Lancet. Neurology
DO  - 10.1016/s1474-4422(23)00313-7
UR  - https://doi.org/10.1016/s1474-4422(23)00313-7
ER  -

APA

PB, S., NL, K., JJ, D., W, M., CG, B., AM, S., L, S., BK, S., F, M., A, B., AR, F., DN, S., S, N., LN, A., AH, B., A, B., MK, C., T, D., RJ, G., M, J., J, C., V, M., ES, J., J, L., F, M., W, M., F, V., M, M., C, H., M, N., MW, L., S, P., & S, R. (2023). Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.. The Lancet. Neurology. https://doi.org/10.1016/s1474-4422(23)00313-7

Source records

pubmed · retrieved 2026-09-26T08:22:46.689Z