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​Dana-Dwek Children’s Hospital was the first in Israel to administer the FDA-approved drug Sprinraza for treating spinal muscular atrophy (SMA) in infants. The patient, a six-month-old girl who responded well to her first dose of the therapy, is one of the 6,000 to 11,000 people afflicted with this genetic condition. Without treatment, patients lose motor neuron function and experience progressive muscle atrophy, especially in the respiratory system, which can lead to premature death.

A breakthrough toward a cure

Prof. Aviva Fattal-Valevski, head of the hospital’s Pediatric Neurology Unit, said, “This is a breakthrough in the treatment of genetic diseases of this type, which in the past would have been considered science fiction.”
We hope that the drug will soon become part of the health basket, joining the list of medications available to patients in Israel.

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