Results 201 to 210 of about 8,917 (217)

Defects in calcium homeostasis and mitochondria can be reversed in Pompe disease

Autophagy, 2015
Jeong-A Lim, Lishu Li, Nina Raben
exaly  

Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder—murine Pompe disease

Autophagy, 2010
Nina Raben, Masaaki Komatsu, Evelyn Ralston   +2 more
exaly  

The Natural Course of Infantile Pompe's Disease: 20 Original Cases Compared With 133 Cases From the Literature

Pediatrics, 2003
Arnold J J Reuser, Ans T Van Der Ploeg
exaly  

A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

New England Journal of Medicine, 2010
Ans T Van Der Ploeg, Paula R Clemens, Geert Jan Groeneveld   +2 more
exaly  

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

Molecular Genetics and Metabolism, 2010
Priya S Kishnani, Deeksha Bali, Dwight D Koeberl   +2 more
exaly  

Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review

Journal of Neurology, 2012
Benedikt Schoser
exaly  

Pompe disease: from pathophysiology to therapy and back again

Frontiers in Aging Neuroscience, 2014
Jeong-A Lim, Lishu Li, Nina Raben
exaly  

European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10‐year experience

European Journal of Neurology, 2017
Ans T Van Der Ploeg
exaly  

Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease

Pediatric Research, 2009
Priya S Kishnani, Deya Corzo, Ans T Van Der Ploeg   +2 more
exaly  

The emerging phenotype of late-onset Pompe disease: A systematic literature review

Molecular Genetics and Metabolism, 2017
Zoheb B Kazi, Laura E. Case, Priya S Kishnani   +2 more
exaly