Results 141 to 150 of about 135,809 (156)

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  

Enfermedad de Pompe del adulto: análisis de 13 pacientes

Medicina Clínica
Paloma Martín-Jiménez, Laura Bermejo-Guerrero, Ana Hernandez-Voth, Ana Arteche-López, Aurelio Hernández-Lain, María Rabasa, Cristina Domínguez-González   +6 more
openaire   +1 more source

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  

Variabilidad en la presentación clínica en la enfermedad de Pompe:: evolución tras terapia de reemplazo enzimático

, 2015
M. Martos, M. Reyes, R. E. Rosso, J. Solera, L. M. Jiménez   +4 more
semanticscholar   +1 more source

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  

Síntomas atípicos compatibles con fibromialgia dilatan el diagnóstico en paciente con enfermedad de Pompe de inicio tardío

, 2017
E. Calabrese
semanticscholar   +1 more source