All Orphan Drugs articles
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NewsNew organoid model helps test spinal cord regeneration drugs
Northwestern scientists have grown human spinal cord organoids to test therapies that could reduce scarring and promote nerve regrowth in patients.
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NewsKidney fibrosis linked to overactive Hippo signalling pathway
Scientists have used human stem cell–derived kidney organoids to uncover how abnormal Hippo signalling drives scarring in nephronophthisis, a rare genetic kidney disease.
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ArticleThe mother of invention: from steam engines to AI-designed drugs
Every great leap in history started with a single, urgent need. Now AI is emerging as the next great engine of invention, transforming the future of medicine faster than ever imagined.
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NewsGlioblastoma treatment boosted by new drug combination strategy
Researchers in China have discovered how glioblastoma adapts to low-oxygen conditions – revealing a new treatment strategy that combines an orphan drug with standard chemotherapy to overcome resistance.
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NewsRare disease drugs get boost from new NICE guidelines
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.
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NewsNew partnership targets CNS symptoms in ultra-rare diseases
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.
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NewsNew therapy for incurable brain disease heads to clinical trials
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.
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ArticleNew tRNA tech aims to rewrite rare disease treatment
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.
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NewsNew UCD centre targets the biggest bottleneck in rare research
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.
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ArticleDriving 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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ArticleGene 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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NewsITM-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.