Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study

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Location: Deutsche Nationalbibliothek Frankfurt am Main

ISSN: 1750-1172

Extent: Online-Ressource

Language: Englisch

Notes: online resource.

Bibliographic citation: Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study ; volume:8 ; number:1 ; day:27 ; month:3 ; year:2013 ; pages:1-7 ; date:12.2013
Orphanet journal of rare diseases ; 8, Heft 1 (27.3.2013), 1-7, 12.2013

Creator: Güngör, Deniz

Contributor: Kruijshaar, Michelle E.
Plug, Iris
D’Agostino, Ralph B.
Hagemans, Marloes LC
van Doorn, Pieter A.
Reuser, Arnold JJ
van der Ploeg, Ans T.
SpringerLink (Online service)

DOI: 10.1186/1750-1172-8-49

URN: urn:nbn:de:1111-2016050122487

Rights: Der Zugriff auf das Objekt ist unbeschränkt möglich.

Last update: 14.08.2025, 10:52 AM CEST

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Associated

Güngör, Deniz
Kruijshaar, Michelle E.
Plug, Iris
D’Agostino, Ralph B.
Hagemans, Marloes LC
van Doorn, Pieter A.
Reuser, Arnold JJ
van der Ploeg, Ans T.
SpringerLink (Online service)

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

Enzyme replacement therapy (ERT) in pompe disease

Enzyme replacement therapy and fatigue in adults with Pompe disease

Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

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

Hochschulschrift

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

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

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

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

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

Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy

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

Enzyme replacement therapy (ERT) in pompe disease

Enzyme replacement therapy and fatigue in adults with Pompe disease

Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

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

Hochschulschrift

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

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

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

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

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

Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy

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Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study

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Other Objects (12)

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

Enzyme replacement therapy (ERT) in pompe disease

Enzyme replacement therapy and fatigue in adults with Pompe disease

Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

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

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

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

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

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

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

Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy

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

Enzyme replacement therapy (ERT) in pompe disease

Enzyme replacement therapy and fatigue in adults with Pompe disease

Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

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

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

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

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

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

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

Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy

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

Enzyme replacement therapy (ERT) in pompe disease

Enzyme replacement therapy and fatigue in adults with Pompe disease

Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

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

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

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

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

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

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

Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy

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