Guided by the Department of Health – Abu Dhabi (DoH), Burjeel Medical City (BMC) has administered Givosiran, an innovative treatment for a rare genetic disorder that leads to the buildup of toxic metabolites in the liver, for the first time in the UAE, marking a significant milestone for healthcare in the country.
Muhammad, a 21-year-old Emirati diagnosed with Acute Intermittent Hepatic Porphyria (AIP), a rare condition that affects only five in one million people, was the first patient in the UAE to receive the Givosiran medication at BMC. AIP, caused by a defective enzyme, leads to the accumulation of toxic metabolites in the liver. These metabolites trigger symptoms such as neuropsychiatric disorders, and can result in life-threatening complications, including renal failure, paralysis, liver failure, and liver cancer.
Dr Asma Al Mannaei, the Executive Director of the Health Life Science Sector at the Department of Health – Abu Dhabi (DoH), said: “Abu Dhabi is dedicated to delivering the highest quality of care and enhancing the health and well-being of our community. Integrating the latest health technology innovations reflects the commitment of all stakeholders to position Abu Dhabi as a leading global healthcare destination.
“The Department’s Research and Innovation Centre’s multidisciplinary team thoroughly evaluated, coded and documented the medication with an identification code. This process enables healthcare facilities in the Emirate to efficiently provide the medication and ensures its inclusion in the health insurance network, streamlining access to the treatment. Administered as a monthly injection, this innovative treatment, utilises small interference RNA technology to effectively reduce the levels of toxic metabolites in the body.”
Dr Niyas Khalid, Specialist for Internal Medicine at BMC, said: “Muhammad’s case represents a significant milestone in treating rare diseases in the UAE. Givosiran is now officially available on the list of approved drugs in the country, paving the way for more patients to access this treatment. This case demonstrates BMC’s prowess in rare diseases and complex care. Some genetic diseases can have very uncommon presentations. But with a high level of suspicion and expertise, it is possible to decode them. “
Muhammad’s mother said: “Our son battled a rare health condition for years, with symptoms such as severe abdominal pain, constant fatigue, weight loss, and frequent hospitalisations. Meeting Dr Khalid at Burjeel Medical City (BMC) marked a turning point, his persistence and expertise led to the correct diagnosis. I am grateful to the Department of Health – Abu Dhabi (DoH) for facilitating the treatment for my son in record time.
“After Muhammad’s first dose of Givosiran, we noticed a significant decrease in his symptoms. We thank our wise leadership for their unwavering support in providing modern treatments and the medical team at BMC.”
The medical team at BMC continues to monitor Muhammad’s progress, aiming for Givosiran to significantly improve his quality of life and prevent further complications. An ENVISION clinical study demonstrated a 74 per cent reduction in annualised symptom rates, a 90 per cent reduction in ALA and PBG levels, and a significant improvement in patients’ quality of life.
