Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma

gene therapy orphan drug pharmacovigilance safety data spinal muscular atrophy

Journal

Pharmaceuticals (Basel, Switzerland)
ISSN: 1424-8247
Titre abrégé: Pharmaceuticals (Basel)
Pays: Switzerland
ID NLM: 101238453

Informations de publication

Date de publication:
19 Mar 2024
Historique:
received: 26 02 2024
revised: 13 03 2024
accepted: 15 03 2024
medline: 28 3 2024
pubmed: 28 3 2024
entrez: 28 3 2024
Statut: epublish

Résumé

The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma

Identifiants

DOI: 10.3390/ph17030394 PMID: 38543180
pubmed: 38543180
pii: ph17030394
doi: 10.3390/ph17030394
pii:
doi:

Types de publication

Journal Article

Langues

eng

Auteurs

Rosanna Ruggiero (R)

Department of Experimental Medicine, University of Campania "Luigi Vanvitelli", 80138 Naples, Italy.
Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy.

Nunzia Balzano (N)

Department of Experimental Medicine, University of Campania "Luigi Vanvitelli", 80138 Naples, Italy.
Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy.

Maria Maddalena Nicoletti (MM)

Department of Precision Medicine, University of Campania "Luigi Vanvitelli", 80138 Naples, Italy.

Gabriella di Mauro (G)

Department of Experimental Medicine, University of Campania "Luigi Vanvitelli", 80138 Naples, Italy.
Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy.
UOC Pharmacy, AORN Santobono-Pausilipon Children's Hospital, 80122 Naples, Italy.

Federica Fraenza (F)

Department of Experimental Medicine, University of Campania "Luigi Vanvitelli", 80138 Naples, Italy.
Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy.

Maria Rosaria Campitiello (MR)

Department of Obstetrics and Gynaecology and Physiopathology of Human Reproduction, ASL Salerno, 84124 Salerno, Italy.

Francesco Rossi (F)

Department of Experimental Medicine, University of Campania "Luigi Vanvitelli", 80138 Naples, Italy.
Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy.
Department of Life Sciences, Health and Health Professions, Link Campus University, 00165 Rome, Italy.

Annalisa Capuano (A)

Department of Experimental Medicine, University of Campania "Luigi Vanvitelli", 80138 Naples, Italy.
Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy.

Classifications MeSH