Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report
Priya S. Kishnani(Duke Medical Center), Yin‐Hsiu Chien(National Taiwan University Hospital), Catherine Wilson(Murdoch Children's Research Institute), S. Grace Prakalapakorn(Duke University), James Davison(Great Ormond Street Hospital for Children NHS Foundation Trust), Satoko Kumada(Tokyo Metropolitan Neurological Hospital), Xianzhang Meng(Sanofi (United States)), Kristina An Haack(Sanofi (France)), Alexander Broomfield(St Mary's Hospital), Hirotaka Ohki(Tokyo Metropolitan Children's Medical Center), Susan Sparks(Children's National), Si Houn Hahn(University of Washington), Anaïs Brassier(Hôpital Necker-Enfants Malades), Barbara Kittner(Sanofi (United States)), David Kronn(New York Medical College), Samia Pichard(Hôpital Necker-Enfants Malades), François Labarthe(Université de Tours), Atef Zaher(Sanofi (United States))
Cited by 53
Related Papers
Biparental Inheritance of Mitochondrial DNA in Humans
|Proceedings of the National Academy of Sciences|2018|451
Glycogen Storage Disease Type III diagnosis and management guidelines
|Genetics in Medicine|2010|305
Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease
|Genetics in Medicine|2009|292
Schwannomatosis
|Neurology|1996|244
Targeted long-read sequencing identifies missing disease-causing variation
|The American Journal of Human Genetics|2021|239