Rare diseases

We fund pioneering research into very rare childhood diseases that can have a devastating impact on children and their families
Project title
Condition, location, fund awarded
X-linked lymphoproliferative syndrome (XLP), University of Zurich, £50,000
Neuroblastoma, Brunel University, £106,941
Cystic fibrosis, University College London, £150,000 (co-funded by the Cystic Fibrosis Trust)
Brittle bone disease, Sheffield Children's Hospital, £25,800
Batten disease, Cardiff University, £16,200
Batten disease, King's College London, £50,000
Ataxia-telangiectasia, Goethe University, €119,450
Myotubular myopathy, The Institute of Genetics & Cellular & Molecular Biology (IGBMC), France, £132,000
Krabbe disease, University of Cambridge, £148,278
Alagille syndrome, University of Cambridge, £223,080