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.