Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline
David Kronn(New York Medical College), Priya S. Kishnani(Duke Medical Center), 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), Swathi Tammireddy, 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), Xianzhang Meng(Jilin Agricultural University), Tianyue Zhou, Atef Zaher(Sanofi (United States)), François Labarthe(Université de Tours), Yin‐Hsiu Chien(National Taiwan University Hospital)
Cited by 1
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