World’s most expensive gene therapy
The compassionate gesture will provide the little girl a complete reversal of her genetic condition with what is the world’s most expensive gene therapy worth Dh8 million.
The toddler, Laveen Jabbar Al Kutyashi, was diagnosed with Stage II Spinal Muscular Dystrophy (SMA), a genetic condition that delays motor development. Her condition would have progressively deteriorated without this one–time life-saving injection that can cure her SMA and reverse the symptoms so far.
Mother in desperate appeal
The parents of the little child cannot thank Sheikh Mohammed enough for covering the cost of treatment of their beloved daughter. Shaikh Mohammad was moved by the plight of the child when he watched a video posted by Laveen’s mother on social media appealing for help.