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NICE has raised the cost-effectiveness thresholds for ultra-rare disease drugs under its Highly Specialised Technology programme – a move welcomed by patient advocates and biotech leaders.
Chiesi Group and Key2Brain have signed a global license agreement to develop two blood-brain barrier-crossing enzyme replacement therapies for alpha-mannosidosis and Krabbe disease – with the aim to help patients with limited treatment options.
SynaptixBio has selected its lead drug candidate, SB H-19642, for clinical trials to treat H-ABC - a rare, fatal neurodegenerative disease with no current cure.
Alltrna is redefining the future of rare disease treatment with engineered tRNA therapeutics that target genetic mutations - not just individual diseases. CEO Michelle Werner shares how this bold, mutation-driven approach could unlock universal treatments for thousands of patients long overlooked by traditional medicine.
The world’s first translational research center dedicated to urea cycle disorders (UCDs) has been launched in Zürich, marking a significant moment in rare disease innovation.
Judy Ashworth, CMO of Novadip Biosciences, shares her journey in advancing treatments for rare paediatric conditions and championing diversity in STEM.
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...
