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The Lancet. Neurology|February 4, 2018
Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic managementDavid J Birnkrant, Katharine Bushby, Carla M Bann, et al.
The Lancet. Neurology|February 4, 2018
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional managementDavid J Birnkrant, Katharine Bushby, Carla M Bann, et al.
Stem Cells Translational Medicine|October 29, 2017
Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled TrialJessica M Sun, Allen W Song, Laura E Case, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 22, 2023
Phase I study of liver depot gene therapy in late-onset Pompe diseaseEdward C Smith, Sam Hopkins, Laura E Case, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 21, 2018
Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe DiseaseDwight D Koeberl, Laura E Case, Edward C Smith, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|July 16, 2010
Glycogen storage disease type III diagnosis and management guidelinesPriya S Kishnani, Stephanie L Austin, Pamela Arn, et al.
Molecular Genetics and Metabolism|February 16, 2023
Diagnosis and management of glycogen storage disease type IV, including adult polyglucosan body disease: A clinical practice resourceRebecca L Koch, Claudia Soler-Alfonso, Bridget T Kiely, et al.
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