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First New Therapy Approved for X-Linked Hypophosphataemia Patients in 30 Years

Vitamin D Resistant Rickets treatment

NHS has paved way for the children and young adults in England and Wales with the rare bone disorder X-linked hypophosphataemia to have access to a new treatment option. This groundbreaking step has been the first significant ray of hope for these patients in the past 30 years. The National Institute for Health and Care Excellence has given a nod to the use of 'Burosumab' for treating X-linked hypophosphataemia (XLH) in children and young people with growing bones. Burosumab is an anti-FGF23 fully human monoclonal antibody, and has the distinction of being the first treatment to target the pathophysiology of X-linked hypophosphataemia.

XLH is an inherited genetic disorder that causes low levels of phosphate in the blood. It gives rise to soft and weak bones, and affected patients may suffer with bowed legs, bone pain, short stature, delayed walking, dental problems and hearing loss. Current treatment mainly relies on supplementation of vitamin D and oral phosphate. An injection of Burosumab is to be given every 2 weeks from the age of one until the complete growth of skeleton has taken place. This therapy is targeted to increase phosphate reabsorption from the kidney and improve intestinal absorption of both calcium and phosphate. NICE's approval for the drug is a substantial step in the treatment as it is aimed at rectifying the underlying disease mechanism and mitigate physical limitations and emotional challenges.

Complete article can be read here