Emirati girl with spinal muscular atrophy takes her first few steps at Abu Dhabi long-term care hospital

Emirati girl with spinal muscular atrophy takes her first few steps at Abu Dhabi long-term care hospital

Abu Dhabi: A two-year-old Emirati girl who last year received an advanced gene replacement therapy to treat her spinal muscular atrophy (SMA) has recently taken her first few steps in the capital. Afra is one of a number of children in the UAE who is benefiting from a raft of new and comprehensive treatments to treat SMA, a progressive and debilitating neuromuscular disease that can be fatal if untreated. Under treatment at Salma Children’s Rehabilitation Hospital, a long-term post-acute care facility in the network of public health provider, the Abu Dhabi Health Services Company (Seha), Afra has even taken a few sips of water already. Her caregivers are now confident that she will continue to improve with the correct interventions. Diagnosed with SMA soon after birth, Afra received the gene therapy when she was 17 months old at the Sheikh Khalifa Medical City, a tertiary referral hospital in the Seha network. The therapy, which is prohibitively expensive for many, was imported for her benefit. She was then transferred to Salma Children’s Hospital three months later. “When Afra came to us, she was still bedbound, and required breathing support. With regular physical therapy, we have been able to wean her off