Establishing Treatment Effectiveness in Fabry Disease: Observation-Based Recommendations for Improvement.

Fabry Disease / therapy Humans Enzyme Replacement Therapy / methods alpha-Galactosidase / genetics Treatment Outcome Female 1-Deoxynojirimycin / analogs & derivatives Male Isoenzymes / genetics Recombinant Proteins / therapeutic use
Fabry disease chaperone therapy clinical outcomes enzyme replacement therapy (ERT) treatment effectiveness

Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
09 Sep 2024
Historique:
received: 02 08 2024
revised: 02 09 2024
accepted: 07 09 2024
medline: 14 9 2024
pubmed: 14 9 2024
entrez: 14 9 2024
Statut: epublish

Résumé

Fabry disease (FD, OMIM #301500) is caused by pathogenic

Identifiants

DOI: 10.3390/ijms25179752 PMID: 39273698
pubmed: 39273698
pii: ijms25179752
doi: 10.3390/ijms25179752
pii:
doi:

Substances chimiques

alpha-Galactosidase EC 3.2.1.22
migalastat C4XNY919FW
agalsidase alfa 2HLC17MX9G
1-Deoxynojirimycin 19130-96-2
agalsidase beta RZD65TSM9U
Isoenzymes 0
Recombinant Proteins 0

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Auteurs

Bram C F Veldman (BCF)

Department of Endocrinology and Metabolism, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.

Daphne H Schoenmakers (DH)

Department of Endocrinology and Metabolism, Platform "Medicine for Society", Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.
Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma's Children's Hospital, Amsterdam UMC Location Vrije Universiteit, 1081 HV Amsterdam, The Netherlands.
ORCID: 0000-0003-1601-7379

Laura van Dussen (L)

Department of Endocrinology and Metabolism, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.
ORCID: 0000-0002-6539-531X

Mareen R Datema (MR)

Department of Endocrinology and Metabolism, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.

Mirjam Langeveld (M)

Department of Endocrinology and Metabolism, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.
ORCID: 0000-0002-9934-6831

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female

Evaluation of Low-Value Services Across Major Medicare Advantage Insurers and Traditional Medicare.

Ciara Duggan, Adam L Beckman, Ishani Ganguli et al.
1.00
Humans United States Aged Cross-Sectional Studies Medicare Part C

Effectiveness of Virtual Yoga for Chronic Low Back Pain: A Randomized Clinical Trial.

Hallie Tankha, Devyn Gaskins, Amanda Shallcross et al.
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH

questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Maladies lysosomiales › Maladies neurologiques de surcharge lysosomiale › Sphingolipidoses › Maladie de Fabry › Establishing Treatment Effectiveness in Fabry...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Establishing Treatment Effectiveness in Fabry...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Thérapie enzymatique › Thérapie enzymatique substitutive › Establishing Treatment Effectiveness in Fabry...
questionsmedicales.fr › Enzymes et coenzymes › Enzymes › Hydrolases › Glycosidases › Galactosidases › alpha-Galactosidase › Establishing Treatment Effectiveness in Fabry...
questionsmedicales.fr › Qualité, accès, évaluation des soins de santé › Qualité des soins de santé › Mécanismes d'évaluation des soins de santé › Évaluation des résultats et des processus en soins de santé › Évaluation de résultat (soins) › Résultat thérapeutique › Establishing Treatment Effectiveness in Fabry...
questionsmedicales.fr › Glucides › Iminosucres › Iminopyranoses › 1-Désoxynojirimycine › Establishing Treatment Effectiveness in Fabry...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Isoformes de protéines › Isoenzymes › Establishing Treatment Effectiveness in Fabry...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines recombinantes › Establishing Treatment Effectiveness in Fabry...