Violeta Stoyanova‐Beninska

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

Manuel Haas, Viktor Vlcek, Pavel Balabanov et al. · Neuromuscular Disorders · 2014 · 158 citations

Rare Diseases, Urology, Dystrophin Gene +5

Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in Europe

Andreas M. Farkas, Segundo Mariz, Violeta Stoyanova‐Beninska et al. · Frontiers in Medicine · 2017 · 35 citations · Full text

Therapeutic Area, Pharmacotherapy, Incentives Supporting Development +19

Oncologic orphan drugs approved in the EU – do clinical trial data correspond with real-world effectiveness?

Yvonne Schuller, Marieke Biegstraaten, Carla E. M. Hollak et al. · Orphanet Journal of Rare Diseases · 2018 · 22 citations · Full text

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

Gloria M. Palomo, Tomás Pose‐Boirazian, Frauke Naumann‐Winter et al. · Molecular Therapy · 2023 · 13 citations · Full text

Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases

Yvonne Schuller, Carla E. M. Hollak, Christine C. Gispen‐de Wied et al. · Drugs · 2017 · 11 citations · Full text