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Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease.
Koeberl, Dwight D; Case, Laura E; Desai, Ankit; Smith, Edward C; Walters, Crista; Han, Sang-Oh; Thurberg, Beth L; Young, Sarah P; Bali, Deeksha; Kishnani, Priya S.
Affiliation
  • Koeberl DD; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States of America; Department of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC 27710, United States of America. Electronic address: dwight.koeber
  • Case LE; Department of Physical and Occupational Therapy, Duke University School of Medicine, Durham, NC 27710, United States of America.
  • Desai A; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States of America.
  • Smith EC; Division of Neurology, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States of America.
  • Walters C; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States of America.
  • Han SO; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States of America.
  • Thurberg BL; Sanofi Genzyme, Cambridge, MA, USA.
  • Young SP; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States of America.
  • Bali D; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States of America.
  • Kishnani PS; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States of America; Department of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC 27710, United States of America.
Mol Genet Metab ; 129(2): 67-72, 2020 02.
Article in En | MEDLINE | ID: mdl-31839530
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II / Muscle, Skeletal / Albuterol / Late Onset Disorders Type of study: Clinical_trials Limits: Adult / Female / Humans / Male / Middle aged Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2020 Document type: Article Country of publication: Estados Unidos
Fulltext
Add to My VHL
Print
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II / Muscle, Skeletal / Albuterol / Late Onset Disorders Type of study: Clinical_trials Limits: Adult / Female / Humans / Male / Middle aged Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2020 Document type: Article Country of publication: Estados Unidos