NICE Enables Access to First Treatment That Targets the Underlying Pathophysiology of Rare Disease X-Linked Hypophosphataemia (XLH) in Adults1 [Yahoo! Finance]
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Source: Yahoo! Finance
treatment of adults living with X-linked hypophosphataemia (XLH) in England, Wales and Northern Ireland LONDON, June 21, 2024 BUSINESS WIRE )--Adults living with the rare disease X-linked hypophosphataemia (XLH) in England, Wales and Northern Ireland will now have access to the first available treatment to tackle the underlying cause of XLH following NICE's decision to recommend burosumab (marketed as Crysvita) to treat adults who have a confirmed diagnosis of XLH, following a new access deal. This decision ensures equal access to burosumab across the UK, as adults living with XLH in Scotland have been able to access burosumab on the NHS since March 2023, following approval by the Scottish Medicines Consortium (SMC) through their ultra-orphan pathway. Burosumab is also available for eligible children and adolescents in all four UK nations. Initially, NICE published an interim negative decision in its Appraisal Consultation Document (ACD). Following constructive engagement betwee
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