Results 211 to 220 of about 31,724,613 (234)

Recurrent Severe Viral-Induced Rhabdomyolysis Associated With Underlying Genetic Variants in a Young Adult: A Case Report. [PDF]

open access: yesCureus
Stone AM   +13 more
europepmc   +1 more source

GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease. [PDF]

open access: yesMol Ther Adv
Wantuch S   +15 more
europepmc   +1 more source

Consensus guidelines for management of glycogen storage disease type 1b – European Study on Glycogen Storage Disease Type 1

open access: yesEuropean Journal of Pediatrics, 2002
Life expectancy in glycogen storage disease type 1 (GSD-1) has improved considerably. Its relative rarity implies that no metabolic centre has experience of large series of patients and therefore experience with long-term management and follow-up at each
G Peter A Smit, Jan Peter Rake
exaly   +2 more sources

Guidelines for management of glycogen storage disease type I – European Study on Glycogen Storage Disease Type I (ESGSD I)

open access: yesEuropean Journal of Pediatrics, 2002
Life-expectancy in glycogen storage disease type I (GSD I) has improved considerably. Its relative rarity implies that no metabolic centre has experience of large series ofpatients and experience with long-term management and follow-up at each centre
G Peter A Smit, Jan Peter Rake
exaly   +3 more sources
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Liver transplantation for glycogen storage disease type Ia

Journal of Hepatology, 2009
Priya S Kishnani   +2 more
exaly  

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