General Archive
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Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type II
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Phase II Study of a Novel Vitamin Metabolite (CoA-Z) for Pantothenate Kinase Associated Neurodegeneration
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DefINe: A trial to test the use of deferiprone in people with neuroferritinopathy
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The TIARA trial: Investigating a new treatment for a life-threatening blood disorder
The TIARA trial: Investigating a new treatment for a life-threatening blood disorder A collaboration with the Aplastic Anaemia Trust to…
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Innovation Hubs for Gene Therapy: A national network to transform gene therapy research
Innovation Hubs for Gene Therapy: A national network to transform gene therapy research Our partnership with the Medical Research Council…
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GOSH and UCL: Co-funding a new blood test to detect a deadly heart condition in children
GOSH and UCL: Co-funding a new blood test to detect a deadly heart condition in children Researchers at UCL (University…
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GOSH: A partnership to increase access to gene therapies for children with rare diseases
GOSH: A partnership to increase access to gene therapies for children with rare diseases We are collaborating with Great Ormond…
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Funding: Partnering with Great Ormond Street Hospital Charity (GOSH Charity) to deliver translational research accelerator grants
Funding: Partnering with Great Ormond Street Hospital Charity (GOSH Charity) to deliver research grants for paediatric rare diseases With GOSH…
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News releasesPromising results in clinical trial gives hope to patients with blood disorder
A trial for a cutting-edge cell therapy that could become the first ever treatment for the rare, life-threatening blood disorder…
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RAREfest 2024
About the event RAREfest is a unique science, technology, community and arts festival run by Cambridge Rare Disease Network (CamRARE).…
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Getting Cilia Moving
About the event LifeArc are pleased to support this that event will bring together all stakeholders to discuss innovative approaches…
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News releasesResearch shows potential ‘life-transforming’ mole reversal therapy in rare condition
Researchers at the Francis Crick Institute, UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital for…