Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study-Reference-Cited by-同舟云学术

Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study

Published:2020-11-07 Issue:1 Volume:27 Page:129-136
ISSN:1351-8216
Container-title:Haemophilia
language:en
Short-container-title:Haemophilia

Author:

Overbeeke Eline¹^ORCID,Michelsen Sissel¹,Hauber Brett²,Peerlinck Kathelijne³,Hermans Cedric⁴^ORCID,Lambert Catherine⁴^ORCID,Goldman Michel⁵,Simoens Steven¹,Huys Isabelle¹

Affiliation:

1. Clinical Pharmacology and Pharmacotherapy University of Leuven Leuven Belgium

2. Health Preference Assessment RTI Health Solutions Research Triangle Park NC USA

3. Haemophilia Center UZ Leuven Leuven Belgium

4. Haemophilia Clinic St‐Luc University Hospital Brussels Belgium

5. Institute for Interdisciplinary Innovation in healthcare Université libre de Bruxelles Brussels Belgium

Publisher

Wiley

Subject

Genetics(clinical),Hematology,General Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/hae.14190

Reference29 articles.

1. UniQure.UniQure Enrolls First Patient in Phase III HOPE‐B Pivotal Study of AMT‐061 in Patients with Hemophilia B 2018.https://tools.eurolandir.com/tools/Pressreleases/GetPressRelease/?ID=3479015&lang=en-GB&companycode=nl-qure&v=. Accessed June 28 2018

2. Pfizer.Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy 2018.https://www.pfizer.com/news/press-release/press-release-detail/pfizer_initiates_pivotal_phase_3_program_for_investigational_hemophilia_b_gene_therapy-0. Accessed July 16 2018.

3. BioMarin.BioMarin Provides 2 Years of Clinical Data in 6e13 vg/kg Dose from Ongoing Phase 1/2 Study in Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at World Federation of Hemophilia 2018 World Congress 2018.https://investors.biomarin.com/2018-05-22-BioMarin-Provides-2-Years-of-Clinical-Data-in-6e13-vg-kg-Dose-from-Ongoing-Phase-1-2-Study-in-Valoctocogene-Roxaparvovec-Gene-Therapy-for-Severe-Hemophilia-A-at-World-Federation-of-Hemophilia-2018-World-Congress. Accessed May 22 2018

4. Getting Ready: Recommendations for Timely Access to Advanced Therapy Medicinal Products (ATMPs) in Europe. Alliance for Regenerative Medicine (ARM);2019.

5. Modern haemophilia care

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3. The benefits of gene therapy in people with haemophilia;Journal of Viral Hepatitis;2024-04

4. Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study;Orphanet Journal of Rare Diseases;2024-02-10

5. Knowledge and attitudes toward gene therapy of a cohort of Italian patients with hemophilia;Journal of Thrombosis and Haemostasis;2023-12