Gene Therapy in Hemophilia: A Transformational Patient Experience.
clinician-patient relationship
communication
patient engagement
patient perspectives/narratives
patient satisfaction
quality of life
Journal
Journal of patient experience
ISSN: 2374-3735
Titre abrégé: J Patient Exp
Pays: United States
ID NLM: 101688338
Informations de publication
Date de publication:
2023
2023
Historique:
pubmed:
4
9
2023
medline:
4
9
2023
entrez:
4
9
2023
Statut:
epublish
Résumé
Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute to reduced health-related quality of life (HRQoL). The current standard of care is clotting factor replacement, which is associated with regular infusions; therefore, alternative treatments such as gene therapy (GT) are in development. GT involves the delivery of a functional copy of the clotting factor
Identifiants
pubmed: 37663068
doi: 10.1177/23743735231193573
pii: 10.1177_23743735231193573
pmc: PMC10472832
doi:
Types de publication
Journal Article
Langues
eng
Pagination
23743735231193573Informations de copyright
© The Author(s) 2023.