Families from across the UAE marked Rare Disease Day 2022 with a lighting ceremony on Burj Khalifa to build awareness of rare diseases and their impact on patients a.
Abu Dhabi’s Burjeel Hospital provides transformational gene therapy
ABU DHABI, 26 days ago VPS Healthcare’s Burjeel Hospital has successfully administered advanced gene replacement therapy, in an effort to save the lives of seven-month-old Emirati twins. With the gracious support of the government and leaders of the UAE, the therapy has been carried out on the twins by the Abu Dhabi based hospital who diagnosed them with a rare and most severe form of Spinal Muscular Atrophy – Type 1 (SMA1), a highly fatal disease. After visiting Burjeel Hospital for a neurological assessment with Consultant Pediatrician and Child Neurologist, Dr Hussein Matlik, the twin babies were diagnosed with the rare and devastating monogenetic neuromuscular disease, SMA1, which is also known as Werdnig Hoffman disease. SMA is caused by a defective or missing survival motor neuron (SMN1) gene. The genetic disorder affects nerve cells that control voluntary muscles of the body resultin