A BRITISH-INDIAN family is seeking support to help their 18-month-old son, Eliyas, access an investigational treatment in the United States for an ultra-rare genetic condition.
Eliyas has TUBB4A-related leukodystrophy, a neurological disorder that affects the development of myelin, which helps the brain communicate with the rest of the body.
Genetic testing confirmed that he carries a disease-causing variant in the TUBB4A gene. He undergoes physiotherapy to work on movement and developmental milestones.
Eliyas has been accepted for a personalised antisense oligonucleotide (ASO) gene therapy programme at Rush University Medical Center in Chicago.
The treatment is not available through the NHS and must be funded privately. The family is seeking funds for treatment, medical monitoring, physiotherapy, rehabilitation, travel and accommodation.
Donations can be made through the family's GoFundMe appeal.
