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Orphan drugs

news

ITM-11 boosts survival in neuroendocrine tumour trial

3 February 2025 | By Drug Target Review

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.

news

How challenges including Brexit, inadequate funding & insufficient sample sizes hinder rare disease research

4 September 2018 | By SMi Group

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.

news

Paediatric ulcerative colitis drug gets orphan designation

11 August 2017 | By Dr Zara Kassam (Drug Target Review)

The U.S. Food and Drug Administration has granted an orphan-drug designation to cobitolimod for the treatment of ulcerative colitis in pediatric patients...

news

Orphan drug market to double as Pharma rethinks business model

21 July 2017 | By Steve Bremer (Drug Target Review)

Market intelligence suggests pharma is being forced to shift focus towards niche patient populations and orphan diseases ...

Advanced techniques and applications of LC-MS in small molecule drug discovery

news

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...

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Recommended

Orphan drugs

ITM-11 boosts survival in neuroendocrine tumour trial

How challenges including Brexit, inadequate funding & insufficient sample sizes hinder rare disease research

Paediatric ulcerative colitis drug gets orphan designation

Orphan drug market to double as Pharma rethinks business model

Attendee list released for the Orphan Drugs & Rare Diseases 2016