Maria Receives the Most Expensive Drug in the World

22-month-old Maria, from the Bedouin town of Rahat, suffered from a rare genetic disorder called Spinal Muscular Atrophy (SMA), to which her brother had succumbed 5 years ago. She was given the drug Zolgensma, the most expensive in the world, and administered to Maria in the Pediatric Intensive Care Unit at Schneider Children's. The cost was covered by Clalit Health Services.

As Maria will celebrate her second birthday in two months' time, it was urgent to speed up the treatment since the drug is only given up to the age of two years, with the recommendation to give it as early as possible. Maria has been under the care of the Institute of Neurology, headed by Prof. Yoram Nevo, since she was three weeks old. Her treatment and follow-up care is under the supervision of Dr. Sharon Aharoni, senior neurologist in charge of SMA treatment at Schneider Children's.

SMA appears in one in 6,000-10,000 newborns, and results from a mutation in the SMN1 gene that leads to death of the motor neuron cells in the spinal column. These neurons convey signals from the brain to the spinal cord, causing spinal muscles to contract and move the body. Damage to the neurons causes the body to lose physical strength and eventually the ability to walk, speak, swallow and breathe. The drug Zolgensma costs $2.125million for a one-time treatment and is thought be a breakthrough drug for genetic repair. Research has shown that the drug leads to a change in the genetic mutation resulting in a cure from the disease.

Murad, Maria's father, emotionally expressed his gratitude to the doctors at Schneider Children's and Clalit Health Services: "Huge thanks to Schneider Children's and Clalit. Because of them, have reached this moment. I am very excited and have no words to express my gratitude to the devoted staff at Schneider who did and do everything for my daughter. I wish the best of health to all the children."

Schneider Children's recently opened an innovative center for the treatment of rare genetic diseases as part of the Institute of Neurology. Dr. Aharoni stated that "the Neurology Institute treats thousands of children with various rare diseases for which there is no cure. We conducted gene therapy treatment for SMA here for the first time and we are very pleased to have been able to give the drug to Maria. She has a real chance of having a healthy life and we hope that additional innovative research and breakthroughs will be made in the future."