Award success for rare disease research

Approximately 350 million people worldwide are living with a rare disease, and half of them are children. Vital research into lysosomal storage disorders has been recognised for its breakthrough discoveries and potential for impact for patients living with rare diseases.

Dr Helen Waller-Evans, from Cardiff University Medicines Discovery Institute, is one of five UK scientists selected for the Harrington Discovery Institute award for a new UK rare disease program.

The Harrington Discovery Institute at University Hospitals in Cleveland and its registered charity Fund for Cures UK, together with Morgan Stanley GIFT Cures, announced its winners in the inaugural Harrington UK Rare Disease Scholar Award competition to advance novel treatments for rare diseases.

“Rare diseases represent one of the greatest unmet needs in healthcare today. There are more than 7,000 known rare diseases, with only five percent of those having an approved treatment. These rare diseases, such as lysosomal storage disorders, represent a huge unmet medical need.

“The research in my laboratory focusses on characterising the function of potential neuroscience drug targets and assessing the effects of novel drugs on this function. We have a particular drive to develop therapies that act on lysosomal proteins for use in psychiatric and neurological diseases,” said Dr Helen Waller-Evans from Cardiff University’s Medicines Discovery Institute.

The Fund for Cures UK was awarded to Dr Waller-Evan’s research by the Harrington Discovery Institute, in recognition for its potential to progress into clinic. The award provides grand-funding and dedicated drug discovery and development support from Harrington Discovery Institute’s Therapeutics Development team.

“This award will allow us to continue our vital work progressing new therapies for rare diseases,” added Dr Waller-Evans.