Challenging times: Delivering gene therapies and an opportunity for shared learning-Reference-Cited by-同舟云学术

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Challenging times: Delivering gene therapies and an opportunity for shared learning

Published:2021-11-08 Issue: Volume: Page:
ISSN:0306-5251
Container-title:British Journal of Clinical Pharmacology
language:en
Short-container-title:Br J Clin Pharmacol

Author:

Kennedy Cormac¹²^ORCID,O'Dea Edel²,Reidy Derval²,Towns Jeremy²,Pigott Maria²,Kevans David²³,O'Rourke Declan⁴,Hennessy Martina¹²

Affiliation:

1. Department of Pharmacology and Therapeutics, Health Sciences Centre Trinity College Dublin Ireland

2. Wellcome‐HRB Clinical Research Facility St James Hospital Dublin Ireland

3. Department of Gastroenterology St James Hospital Dublin Ireland

4. Department of Neurology Children's Health Ireland at Temple Street Dublin Ireland

Publisher

Wiley

Subject

Pharmacology (medical),Pharmacology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/bcp.15125

Reference8 articles.

1. Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

2. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

3. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

4. Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes

5. Spinal Muscular Atrophy in the Treatment Era

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