BMC, guided by DoH, administers innovative treatment for rare liver disease

Photo: Abu Dhabi Media Office

Burjeel Medical City (BMC), in partnership with the Department of Health – Abu Dhabi (DoH), has made a significant step in healthcare by offering Givosiran, an innovative treatment for a rare genetic liver disorder, for the first time in the UAE. This marks an important milestone for healthcare in the country.

The first patient to receive this treatment in the UAE was 21-year-old Emirati Muhammad, who has been diagnosed with Acute Intermittent Hepatic Porphyria (AIP). AIP is an extremely rare condition that affects just five in one million people. It is caused by a defective enzyme, leading to a buildup of toxic substances in the liver. These toxins can cause serious health problems, including neuropsychiatric disorders and potentially life-threatening issues like kidney failure, paralysis, liver failure, and even liver cancer.

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Dr. Asma Al Mannaei, the Executive Director of the Health Life Science Sector at DoH, emphasized the importance of this achievement, stating, “Abu Dhabi is committed to providing the highest quality healthcare and improving the well-being of our community. The integration of advanced health technologies reflects our dedication to making Abu Dhabi a leading healthcare destination globally.”

The Department’s Research and Innovation Centre played a key role in ensuring that Givosiran was evaluated, documented, and approved for use in Abu Dhabi. This process also helped make the treatment accessible through the local health insurance network, allowing more patients to benefit from it. Givosiran works by using small interference RNA technology to lower the levels of toxic metabolites in the body and is administered as a monthly injection.

Dr. Niyas Khalid, Specialist in Internal Medicine at BMC, shared that Muhammad's case represents a major achievement in treating rare diseases in the UAE. "This is a big step forward," he said. "With Givosiran now officially approved in the country, more patients will have access to this life-changing treatment. BMC’s expertise in handling rare diseases and complex cases makes us proud to be part of this journey.”

Muhammad’s mother expressed her gratitude, saying, “For years, my son suffered from severe abdominal pain, constant fatigue, weight loss, and frequent hospitalizations. Finding the right diagnosis was a challenge. But after meeting Dr. Khalid at BMC, everything changed. His expertise led to the right diagnosis, and we are thankful to the Department of Health – Abu Dhabi for making this treatment available to us so quickly.”

After Muhammad received his first dose of Givosiran, his symptoms showed a noticeable improvement. His mother added, “We saw a significant decrease in his symptoms. We are thankful to the leadership for their support in providing modern treatments, and we appreciate the medical team at BMC for their care.”

The medical team at BMC continues to monitor Muhammad’s condition closely. They hope Givosiran will improve his quality of life and help prevent future complications. A recent clinical study called ENVISION showed that Givosiran led to a 74% reduction in annual symptoms, a 90% drop in harmful metabolites, and a big improvement in patients’ quality of life.

This breakthrough marks a new chapter in the treatment of rare genetic disorders in the UAE, making advanced care more accessible to those who need it the most.

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