Adults in England with the rare genetic condition XLH (X-linked Hypophosphatemia) can now access a revolutionary treatment on the NHS. Burosumab, a targeted therapy, addresses the underlying cause of the condition by stabilising phosphate levels in the blood, which are essential for healthy bones and teeth. This breakthrough treatment reduces pain, improves bone health, and enhances overall quality of life for those affected by XLH.
Previously, treatment options were limited to phosphate supplements and activated vitamin D, which often caused significant side effects such as kidney stones and gastrointestinal issues. Burosumab offers a more effective alternative by suppressing the hormone responsible for low phosphate levels, enabling better bone healing and reducing the risk of fractures and surgeries. Patients will also be trained to administer the therapy at home, reducing the need for hospital visits.
This therapy marks a significant step forward for adults with XLH, providing relief from debilitating symptoms such as bone softening, muscle weakness, and dental complications. By improving mobility and reducing reliance on painkillers, Burosumab is set to transform the lives of many individuals living with this challenging condition.
The NHS roll-out follows approval from NICE, which deemed the treatment clinically and cost-effective. Advocacy groups have welcomed this development, emphasising its potential to significantly reduce the physical and emotional burden of XLH.