UAE: New drug saves 21-year-old with rare disease who once 'struggled to get out of bed'

Khaleej Times

26-02-2025

For the last four years, 21-year-old Muhammad, an Emirati student, battled a mysterious illness that left him in pain and fatigue. What started as occasional discomfort soon became a suffering for him with diarrhea, nausea, vomiting, palpitations, and exhaustion. His mother, Fathima, watched as her son's health declined.
'At first, his symptoms weren't frequent,' said Fathima. 'He would feel discomfort after eating, sometimes have diarrhea, and then nausea. But over time, it became worse. Even mild activities left him exhausted, and he needed to rest all the time.'
For years, Muhammad and his family sought medical help, treating his symptoms as they appeared. But the episodes kept coming back and sometimes once or twice a week. 'We did not have any answers for his condition.'
'Then, in the last two years, his condition worsened. The attacks became more frequent, forcing them to rush him to the hospital at least once a month. It began to take a toll on his academics, social life, and daily activities. Some mornings, he struggled just to get out of bed,' said Fathima.
The turning point came last year when they met Dr Niyas Khalid at Burjeel Medical City (BMC). After careful evaluation, Muhammad was diagnosed with Acute Intermittent Hepatic Porphyria (AIP), a rare genetic disorder affecting the liver. The disease, which impacts only five in one million people, causes a buildup of toxic metabolites, leading to severe complications such as neuropsychiatric disorders, renal failure, paralysis, and even liver cancer.
Fathima was shocked but relieved to finally have an answer. 'We had been searching for so long. It was a difficult journey, but now we knew what we were dealing with,' she said.
With the support of the Department of Health (DoH), Muhammad became the first patient in the UAE to receive Givosiran, an innovative treatment designed for AIP patients. The drug is administered as an injection every month. The medication works by reducing toxic metabolite levels, preventing further attacks and complications. The Department of Health's Research and Innovation Centre ensured the medication's approval and inclusion in the health insurance network, making it more accessible for those in need.
After his first dose, the results were remarkable. 'We noticed a significant decrease in his attacks,' Fathima said. 'Now, he is almost living a nearly normal life.'
Dr Niyas Khalid, who led Muhammad's treatment, highlighted the worthiness of this medical breakthrough. 'His case represents a milestone in treating rare diseases in the UAE. With Givosiran now available in the country, more patients will have access to life-changing treatment,' he said.
Muhammad is now continuing his treatment and his family remain hopeful for a healthier future. 'He will need the medication for life, but Inshallah, he will keep improving,' said Fathima.