A real-world analysis of the impact of X-linked myotubular myopathy on caregivers in the United States

A real-world analysis of the impact of X-linked myotubular myopathy on caregivers in the United States

Abstract Background X-linked myotubular myopathy (XLMTM) is a rare, life-threatening congenital myopathy with multisystem involvement, which often includes the need for invasive ventilator support, gastrostomy tube feeding, and wheelchair use in approximately 80% of patients. The direct and indirect...

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Bibliographic Details
Main Authors: Tina Duong, Tmirah Haselkorn, Beckley Miller, Julie Coats, Ivar Jensen, Erin Ward, Marie Wood, Robert J. Graham, Laurent Servais
Format: Article
Language:English
Published: BMC 2025-05-01
Series:Orphanet Journal of Rare Diseases
Subjects:
XLMTM
Myotubular myopathy
Congenital myopathy
Rare disease
Quality of life
Economic impact
Online Access:https://doi.org/10.1186/s13023-025-03583-w
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https://doi.org/10.1186/s13023-025-03583-w

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