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Comment
. 2023 Dec;22(12):1089-1091.
doi: 10.1016/S1474-4422(23)00416-7.

Gene therapy for X-linked myotubular myopathy: the challenges

Nicol C Voermans  1 , Ana Ferreiro  2 , Annemieke Aartsema-Rus  3 , Heinz Jungbluth  4
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Comment

Gene therapy for X-linked myotubular myopathy: the challenges

Nicol C Voermans et al. Lancet Neurol. 2023 Dec.

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Conflict of interest statement

NCV was Principal Investigator of the Unite-CNM trial on Dynacure, and is a member of the scientific advisory board of ZNM Zusammen Stark!. HJ has acted in an advisory capacity for Audentes and Astellas, two companies involved in the development of treatments for X-linked myotubular myopathy. AAR and AF declare no competing interests. All authors are members of the European Reference Network for rare neuromuscular diseases. The authors are grateful to all patients and their families who participated in the natural history studies and the ASPIRO trial, and to the international patient organisations ZNM Zusammen Stark!, the Myotubular Trust, and the MTM-CNM Family Connection.

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