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Balancing expectations: An unmet need in the orphan disease arena
https://pharmaphorum.com/views-analysis-market-access/balancing-expectations-rare-disease-arena/
rare disease
Only 4 Weeks Until Orphan Drugs & Rare Diseases 2018
Pharma Giants Unite to Improve Rare Disease Treatment
Amicus’ oral Fabry disease drug approved in US
Decision follows approval in Europe
