Levelling up UK rare disease research

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The UK needs new approaches to developing treatments for rare diseases, according to a new report led by UCL researchers describing how universities, the pharmaceutical industry and the NHS could improve life sciences collaborations.

The report authors are calling for an independent British Office for Rare Diseases, comparable to the Office of Rare Disease Research in the US.

Report co-author Professor David Taylor (UCL School of Pharmacy) said: “Better treatments are already becoming available, particularly for more common forms of rare disease. The advent of cell, gene and other advanced therapies promises more gains. But progress will depend on funding early stage research in settings like universities and providing sufficient financial incentives for companies to develop and provide safe and effective licensed medicines.

Reference Link:https://www.ucl.ac.uk/news/2022/may/levelling-uk-rare-disease-research