The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products

Author:

Palomo Gloria M.,Pose-Boirazian Tomas,Naumann-Winter Frauke,Costa Enrico,Duarte Dinah M.,Kalland Maria E.,Malikova Eva,Matusevicius Darius,Vitezic Dinko,Larsson Kristina,Magrelli Armando,Stoyanova-Beninska Violeta,Mariz Segundo

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference17 articles.

1. Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in Europe;Farkas;Front. Med.,2017

2. Gene therapy clinical trials, where do we go? An overview;Arabi;Biomed. Pharmacother.,2022

3. Viral vector platforms within the gene therapy landscape;Bulcha;Sig Transduct Target Ther.,2021

4. Scientific and regulatory considerations for adeno-associated viral vector (AAV)-based gene therapy,2018

5. From Laboratory to Patient: The Journey of a Centrally Authorised Medicine,2019

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