Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis

Volume: 105, Issue: 4, Pages: 332 - 338
Published: Sep 4, 2019
Abstract
Purpose Individuals with X-linked myotubular myopathy (XLMTM) who survive infancy require extensive supportive care, including ventilator assistance, wheelchairs and feeding tubes. Half die before 18 months of age. We explored respiratory support and associated mortality risk in RECENSUS, particularly among patients ≤5 years old who received respiratory support at birth; this subgroup closely matches patients in the ASPIRO trial of gene therapy...
Paper Details
Title
Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis
Published Date
Sep 4, 2019
Volume
105
Issue
4
Pages
332 - 338
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