The effectiveness of enzyme replacement therapy for juvenile-onset Pompe disease: a systematic review

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Location: Deutsche Nationalbibliothek Frankfurt am Main

ISSN: 1573-2665

Extent: Online-Ressource

Language: Englisch

Notes: online resource.

Bibliographic citation: The effectiveness of enzyme replacement therapy for juvenile-onset Pompe disease: a systematic review ; day:5 ; month:6 ; year:2018 ; pages:1-8
Journal of inherited metabolic disease ; (5.6.2018), 1-8

Classification: Medizin, Gesundheit

Creator: Milverton, Joanne

Contributor: Newton, Skye
Merlin, Tracy
SpringerLink (Online service)

DOI: 10.1007/s10545-018-0198-8

URN: urn:nbn:de:101:1-2018081122152139283609

Rights: Der Zugriff auf das Objekt ist unbeschränkt möglich.

Last update: 15.08.2025, 7:24 AM CEST

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Milverton, Joanne
Newton, Skye
Merlin, Tracy
SpringerLink (Online service)

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The effectiveness of enzyme replacement therapy for juvenile-onset Pompe disease: a systematic review

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Other Objects (12)

The impact of interrupting enzyme replacement therapy in late-onset Pompe disease

Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy

Enzyme replacement therapy (ERT) in pompe disease

Clinical and Genetic Aspects of Juvenile Onset Pompe Disease

Improvement of bone mineral density after enzyme replacement therapy in Chinese late-onset Pompe disease patients

Enzyme replacement therapy and fatigue in adults with Pompe disease

Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

Impact of enzyme replacement therapy on survival in adults with Pompe disease

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Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

The impact of interrupting enzyme replacement therapy in late-onset Pompe disease

Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy

Enzyme replacement therapy (ERT) in pompe disease

Clinical and Genetic Aspects of Juvenile Onset Pompe Disease

Improvement of bone mineral density after enzyme replacement therapy in Chinese late-onset Pompe disease patients

Enzyme replacement therapy and fatigue in adults with Pompe disease

Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

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Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease

Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients

Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

The impact of interrupting enzyme replacement therapy in late-onset Pompe disease

Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy

Enzyme replacement therapy (ERT) in pompe disease

Clinical and Genetic Aspects of Juvenile Onset Pompe Disease

Improvement of bone mineral density after enzyme replacement therapy in Chinese late-onset Pompe disease patients

Enzyme replacement therapy and fatigue in adults with Pompe disease

Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

Impact of enzyme replacement therapy on survival in adults with Pompe disease

Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease

Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients

Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

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