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Driving diversity and leadership in the biotech industry
Judy Ashworth, CMO of Novadip Biosciences, shares her journey in advancing treatments for rare paediatric conditions and championing diversity in STEM.
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Orphan drugs
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Gene silencing: a step forward for rare disease therapy
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.
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ITM-11 boosts survival in neuroendocrine tumour trial
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.
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How challenges including Brexit, inadequate funding & insufficient sample sizes hinder rare disease research
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.
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Paediatric ulcerative colitis drug gets orphan designation
The U.S. Food and Drug Administration has granted an orphan-drug designation to cobitolimod for the treatment of ulcerative colitis in pediatric patients...
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Orphan drug market to double as Pharma rethinks business model
Market intelligence suggests pharma is being forced to shift focus towards niche patient populations and orphan diseases ...
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Attendee list released for the Orphan Drugs & Rare Diseases 2016
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...
