Emirati twin infants get gene replacement therapy in Abu Dhabi to correct genetic condition ...United Arab Emirates

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, para

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Emirati twin infants get gene replacement therapy in Abu Dhabi to correct genetic condition ...United Arab Emirates

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