Study on hearing loss in MPS is largest yet
Hearing loss is a common but under-recognised complication of the rare disease MPS (mucopolysaccharidoses, a group of inherited conditions caused...
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MoreFirst patient in UK treated with pioneering new gene therapy for Gaucher disease
The first patient in the UK has been treated with a pioneering new investigational gene therapy designed to treat the...
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MoreHydrotherapy study aims to reduce pain for people with rare syndrome
Metabolic specialists at Salford Royal have launched a new study which aims to reduce joint pain and improve mobility for...
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MorePatient-led charity supports research into rare and incurable genetic condition
A new transatlantic partnership will support research aiming to find an effective treatment for a rare genetic condition. Neurofibromatosis type...