Medicine Development and Access for Rare Diseases: Can We Do Better?罕见病药物研发与可及性:我们能做得更好吗?Journal of Inherited Metabolic DiseaseIF3.82026-02-170OAAI
Application of four pricing models for orphan medicines: a case study for lumasiranORPHANET JOURNAL OF RARE DISEASESIF3.52024-12-230OAAI
Framework for Multistakeholder Patient Registries in the Field of Rare DiseasesNEUROLOGYIF8.52024-09-241OAAI
Effectiveness and safety of mexiletine in patients at risk for (recurrent) ventricular arrhythmias: a systematic reviewEUROPACEIF7.42022-08-2914OAAI
Clinical impact of the worldwide shortage of verteporfin (Visudyne®) on ophthalmic careACTA OPHTHALMOLOGICAIF2.82022-04-0666OA