Enhancing Clinical Infrastructure for the Delivery of Intrathecal and Genetic Therapies: A Qalsody (Tofersen) Model for Patients With


Journal

Neurology. Clinical practice
ISSN: 2163-0402
Titre abrégé: Neurol Clin Pract
Pays: United States
ID NLM: 101577149

Informations de publication

Date de publication:
Aug 2024
Historique:
received: 25 07 2023
accepted: 06 02 2024
pmc-release: 01 08 2025
medline: 10 6 2024
pubmed: 10 6 2024
entrez: 10 6 2024
Statut: ppublish

Résumé

Qalsody (tofersen), an intrathecal therapy (IT) antisense oligonucleotide (ASO), was granted accelerated approval by the Food and Drug Administration for the treatment of We predicted that 6 symptomatic and 14 presymptomatic As new therapies for neurologic diseases come to market, preparing for increased office volume and complex drug delivery are essential for optimal care.

Sections du résumé

Background UNASSIGNED
Qalsody (tofersen), an intrathecal therapy (IT) antisense oligonucleotide (ASO), was granted accelerated approval by the Food and Drug Administration for the treatment of
Recent Findings UNASSIGNED
We predicted that 6 symptomatic and 14 presymptomatic
Implications for Practice UNASSIGNED
As new therapies for neurologic diseases come to market, preparing for increased office volume and complex drug delivery are essential for optimal care.

Identifiants

pubmed: 38855716
doi: 10.1212/CPJ.0000000000200303
pii: CPJ-2023-000388
pmc: PMC11157423
doi:

Types de publication

Journal Article

Langues

eng

Pagination

e200303

Informations de copyright

© 2024 American Academy of Neurology.

Déclaration de conflit d'intérêts

The authors report no relevant disclosures. Full disclosure form information provided by the authors is available with the full text of this article at Neurology.org/cp.

Auteurs

Jennifer Morganroth (J)

Department of Neurology, University of Pennsylvania.

Tanya M Bardakjian (TM)

Department of Neurology, University of Pennsylvania.

Laynie Dratch (L)

Department of Neurology, University of Pennsylvania.

Colin C Quinn (CC)

Department of Neurology, University of Pennsylvania.

Lauren B Elman (LB)

Department of Neurology, University of Pennsylvania.

Classifications MeSH