Twins suffering from rare disease get treatment
01 Mar 2021
Mariecar Jara-Puyod,
Senior Reporter
An Emirati couple in Abu Dhabi is grateful for the support they have received, giving their seven-month old identical twins, found to be suffering from a rare disease, the chance to live and enjoy life.
Rashid Al Hasani and Mona Ali’s sons underwent a one-and-a half hour (10:30 a.m. to 12 noon) infusion of the advanced gene therapy replacement known generically as Onasemnogene Abeparvovec (AVXS-101) on Feb. 24 (Wednesday) at Burjeel Hospital in Abu Dhabi. They had earlier sought consultations with consultant paediatrician/child neurologist Dr. Hussein Matlik who had recommended a genetics test. The test diagnosis was Werdnig-Hoffman Disease.
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Abu Dhabi: A pair of seven-month-old Emirati twins have successfully received advanced gene replacement therapy at an Abu Dhabi hospital to treat their highly fatal genetic condition.
The twins were born with Spinal Muscular Atrophy Type 1 (SMA1), a rare and devastating monogenetic neuromuscular disease, also known as Werdnig Hoffman disease, which is caused by a defective or missing survival motor neuron gene (SMN1). The disorder affects the voluntary muscles of the body, resulting in rapid and irreversible loss of motor neurons, which then affects all muscle functions, including breathing, swallowing and basic movement. Over time, the condition leads to progressive muscle weakness, paralysis and even death if left untreated.
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