Real-world analysis of healthcare resource utilization by patients with X-linked myotubular myopathy (XLMTM) in the United States
- Location
-
Deutsche Nationalbibliothek Frankfurt am Main
- Extent
-
Online-Ressource, 1 online resource.
- Language
-
Englisch
- Bibliographic citation
-
Real-world analysis of healthcare resource utilization by patients with X-linked myotubular myopathy (XLMTM) in the United States ; volume:18 ; number:1 ; day:6 ; month:6 ; year:2023 ; pages:1-11 ; date:12.2023
Orphanet journal of rare diseases ; 18, Heft 1 (6.6.2023), 1-11, 12.2023
- Creator
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Graham, Robert J.
Darras, Basil T.
Haselkorn, Tmirah
Fisher, Dan
Genetti, Casie A.
Miller, Weston
Beggs, Alan H.
- Contributor
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SpringerLink (Online service)
- DOI
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10.1186/s13023-023-02733-2
- URN
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urn:nbn:de:101:1-2023091516051034456351
- Rights
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Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Last update
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14.08.2025, 10:55 AM CEST
Data provider
This object is provided by:
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Associated
- Graham, Robert J.
- Darras, Basil T.
- Haselkorn, Tmirah
- Fisher, Dan
- Genetti, Casie A.
- Miller, Weston
- Beggs, Alan H.
- SpringerLink (Online service)
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X-linked myotubular myopathy is associated with epigenetic alterations and is ameliorated by HDAC inhibition
Missense mutations in small muscle protein X-linked (SMPX) cause distal myopathy with protein inclusions
rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathy
Long term preliminary safety and efficacy outcomes for x-linked myotubular myopathy with gene replacement therapy
Easily misdiagnosed X-linked adrenoleukodystrophy
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Identification of a Novel Intronic Mutation in VMA21 Associated with a Classical Form of X-Linked Myopathy with Autophagy
A Deep Intronic Variant Activates a Pseudoexon in the MTM1 Gene in a Family with X-Linked Myotubular Myopathy
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X-linked myotubular myopathy in Rottweiler dogs is caused by a missense mutation in Exon 11 of the MTM1 gene
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Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy
X-linked myotubular myopathy is associated with epigenetic alterations and is ameliorated by HDAC inhibition
Missense mutations in small muscle protein X-linked (SMPX) cause distal myopathy with protein inclusions
rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathy
Long term preliminary safety and efficacy outcomes for x-linked myotubular myopathy with gene replacement therapy
Easily misdiagnosed X-linked adrenoleukodystrophy
X-linked disorders with cerebellar dysgenesis
Identification of a Novel Intronic Mutation in VMA21 Associated with a Classical Form of X-Linked Myopathy with Autophagy
A Deep Intronic Variant Activates a Pseudoexon in the MTM1 Gene in a Family with X-Linked Myotubular Myopathy
X-linked SCID with a rare mutation
X-linked myotubular myopathy in Rottweiler dogs is caused by a missense mutation in Exon 11 of the MTM1 gene