World’s most expensive gene therapy
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
Dr Mohammad Al Awadhi, CEO of Al Jalila Children’s Speciality Hospital, told Gulf News, “Luckily for the child, when the family came here from Iraq as directed by His Highness Sheikh Mohammed, she was diagnosed at our centre for excellence for genome studies to be suffering from this genetic condition. Al Jalila Children’s Speciality Hospital is the first and only one hospital in the entire region to provide this remarkable gene therapy for SMA.”
Good prognosis
Once the drug is administered, the child will be monitored by paediatiricians and will also be put through a rehabilitation programme. The doctors attending on her are hopeful of a complete recovery.
‘Faith in humanity restored’
Mohammad, 35, a sales executive in Iraq and his wife, Mondal, also 35, are parents to another six-year-old daughter. “When Laveen was born, she was completely fine and had normal movements. However, by the time she was six months, her movements had stopped and we were worried. Doctors in Iraq suspected Stage II SMA. However, it was diagnosed only in October 2019 when we were able to travel to Turkey after the lockdown for the pandemic was lifted. Genetic tests carried out there confirmed our worst fears and we were in great despair. My wife made that desperate appeal for help on social media and imagine our surprise, when such a powerful leader actually made this gesture to help us. My daughter has a new lease of life, thanks to the generosity of Sheikh Mohmmed.”
What is SMA?
Spinal muscular atrophy (SMA) is a genetic disease affecting the central nervous system, peripheral nervous system and voluntary muscle movement (skeletal muscle).
Most of the nerve cells that control muscles are located in the spinal cord, which accounts for the word spinal in the name of the disease. SMA is muscular because its primary effect is on muscles, which do not receive signals from these nerve cells. Atrophy is the medical term for getting smaller, which is what generally, happens to muscles when nerve cells do not stimulate them.
SMA involves the loss of nerve cells called motor neurons in the spinal cord and the condition falls under the category of a motor neuron disease.
Original news source Credit: gulfnews.com
You must be logged in to post a comment Login