Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients
- Location
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Deutsche Nationalbibliothek Frankfurt am Main
- ISSN
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1750-1172
- Extent
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Online-Ressource
- Language
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Englisch
- Notes
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online resource.
- Bibliographic citation
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Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients ; volume:15 ; number:1 ; day:14 ; month:9 ; year:2020 ; pages:1-9 ; date:12.2020
Orphanet journal of rare diseases ; 15, Heft 1 (14.9.2020), 1-9, 12.2020
- Creator
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van den Dorpel, J. J. A.
Poelman, E.
Harlaar, L.
van Kooten, H. A.
van der Giessen, L. J.
van Doorn, P. A.
van der Ploeg, A. T.
van den Hout, J. M. P.
van der Beek, N. A. M. E.
- Contributor
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SpringerLink (Online service)
- DOI
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10.1186/s13023-020-01482-w
- URN
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urn:nbn:de:101:1-2020110219400226885664
- Rights
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Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Last update
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15.08.2025, 7:31 AM CEST
Data provider
This object is provided by:
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Associated
- van den Dorpel, J. J. A.
- Poelman, E.
- Harlaar, L.
- van Kooten, H. A.
- van der Giessen, L. J.
- van Doorn, P. A.
- van der Ploeg, A. T.
- van den Hout, J. M. P.
- van der Beek, N. A. M. E.
- SpringerLink (Online service)
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Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease
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A higher dose of enzyme therapy in patients with classic infantile Pompe disease seems to improve ventilator-free survival and motor function
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Cognitive outcome of classic infantile Pompe patients receiving enzyme therapy
Effects of immunomodulation in classic infantile Pompe patients with high antibody titers
Antibody formation to enzyme therapy in classic infantile Pompe disease: implications of patient age
Chest MRI to diagnose early diaphragmatic weakness in Pompe disease
Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease
Late-onset Pompe disease: what is the prevalence of limb-girdle muscular weakness presentation?
A higher dose of enzyme therapy in patients with classic infantile Pompe disease seems to improve ventilator-free survival and motor function
Enzymersatztherapie bei Patienten mit infantiler Verlaufsform des Morbus Pompe in Deutschland
A Severe Form of Non-Classic Pompe's Disease with Normal Creatinine Kinase Level
Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease
Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy
Infantile Pompe disease with intrauterine onset: a case report and literature review
Cognitive outcome of classic infantile Pompe patients receiving enzyme therapy
Effects of immunomodulation in classic infantile Pompe patients with high antibody titers
Antibody formation to enzyme therapy in classic infantile Pompe disease: implications of patient age
Chest MRI to diagnose early diaphragmatic weakness in Pompe disease
Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease
Late-onset Pompe disease: what is the prevalence of limb-girdle muscular weakness presentation?
A higher dose of enzyme therapy in patients with classic infantile Pompe disease seems to improve ventilator-free survival and motor function
Enzymersatztherapie bei Patienten mit infantiler Verlaufsform des Morbus Pompe in Deutschland
A Severe Form of Non-Classic Pompe's Disease with Normal Creatinine Kinase Level
Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease
Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy