Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
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Published:2024-01
Issue:
Volume:99
Page:104894
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ISSN:2352-3964
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Container-title:eBioMedicine
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language:en
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Short-container-title:eBioMedicine
Author:
Lawlor Michael W., Schoser Benedikt, Margeta Marta, Sewry Caroline A., Jones Karra A., Shieh Perry B., Kuntz Nancy L., Smith Barbara K., Dowling James J., Müller-Felber Wolfgang, Bönnemann Carsten G., Seferian Andreea M., Blaschek Astrid, Neuhaus Sarah, Foley A. Reghan, Saade Dimah N., Tsuchiya Etsuko, Qasim Ummulwara R., Beatka Margaret, Prom Mariah J., Ott Emily, Danielson Susan, Krakau Paul, Kumar Suresh N., Meng Hui, Vanden Avond MarkORCID, Wells Clive, Gordish-Dressman Heather, Beggs Alan H.ORCID, Christensen Sarah, Conner Edward, James Emma S., Lee Jun, Sadhu Chanchal, Miller Weston, Sepulveda Bryan, Varfaj Fatbardha, Prasad Suyash, Rico SalvadorORCID
Subject
General Biochemistry, Genetics and Molecular Biology,General Medicine
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