Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium-Reference-Cited by-同舟云学术

Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium

Published:2022-01 Issue:1 Volume:6 Page:28-37
ISSN:2352-4642
Container-title:The Lancet Child & Adolescent Health
language:en
Short-container-title:The Lancet Child & Adolescent Health

Author:

Ditters Imke Anne Maartje,Huidekoper Hidde Harmen,Kruijshaar Michelle Elisabeth,Rizopoulos Dimitris,Hahn Andreas,Mongini Tiziana Enrica,Labarthe François,Tardieu Marine,Chabrol Brigitte,Brassier Anais,Parini Rossella,Parenti Giancarlo,van der Beek Nadine Anna Maria Elisabeth,van der Ploeg Ans Tjitske,van den Hout Johanna Maria Pieternel,Mengel Eugen,Hennermann Julia,Smitka Martin,Muschol Nicole,Marquardt Thorsten,Marquardt Martina,Thiels Charlotte,Spada Marco,Pagliardini Veronica,Menni Francesca,della Casa Roberto,Deodato Federica,Gasperini Serena,Burlina Alberto,Donati Alice,Pichard Samia,Feillet François,Huet Fréderic,Mention Karine,Eyer Didier,Kuster Alice,Espil Taris Caroline,Lefranc Jérémie,Barth Magalie,Bruel Henri,Chevret Laurent,Pitelet Gaele,Pitelet Catherine,Rivier François,Dobbelaere Dries

Funder

Prinses Beatrix Spierfonds

Publisher

Elsevier BV

Subject

Developmental and Educational Psychology,Pediatrics, Perinatology and Child Health

Reference32 articles.

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3. Recombinant human alpha-glucosidase from rabbit milk in Pompe patients;Van den Hout;Lancet,2000

4. Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk;Van den Hout;Pediatrics,2004

5. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease;Kishnani;Neurology,2007

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3. Avalglucosidase alfa in infantile-onset Pompe disease: A snapshot of real-world experience in Italy;Molecular Genetics and Metabolism Reports;2024-09

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5. Long term survival in patients with classic infantile Pompe disease reveals a spectrum with progressive brain abnormalities and changes in cognitive functioning;Journal of Inherited Metabolic Disease;2024-04-08