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Gene silencing offers a promising approach to treating rare neurological diseases like H-ABC. With clinical trials on the horizon, find out how targeted therapies could bring real hope to patients and families.
A new targeted radiotherapy offers hope for patients with rare neuroendocrine tumours, demonstrating a significant improvement in progression-free survival in a key clinical trial.
SMi Reports: 8th Annual Orphan Drugs and Rare Diseases Conference to bring together pharmaceutical companies, non-profit organisations and orphan drug developers to explore alliances and facilitate progress.
The U.S. Food and Drug Administration has granted an orphan-drug designation to cobitolimod for the treatment of ulcerative colitis in pediatric patients...
Market intelligence suggests pharma is being forced to shift focus towards niche patient populations and orphan diseases ...
13 September 2016 | By SMi
SMi Reports (13.09. 2016, London, UK): Alexion, BioMarin, Chiesi, MHRA, NICE to meet in London in October to discuss updates in the orphan drug landscape...
