Prof. Shimon Reif, head of the Department of Pediatrics at Hadassah Hospital Ein Kerem, successfully advocated for health insurance coverage of a medicine that a two-month-old baby with a rare metabolic disease needed to survive.
The baby’s genetic condition leads to her inability to metabolize fats. As Prof. Reif recently explained on Israeli radio, orphan diseases like this rarely have cures and when they do, the costs are very high because the medicine will only be needed for a small number of patients. The cost of this enzyme therapy called Kanuma is half a million shekels a year.
Appearing in the first weeks of life, the disease, called lysosomal acid lipase deficiency, impacts all bodily systems. The child experiences digestive and liver problems and fails to grow and is expected to live only a few months without the medication.
The Jerusalem baby’s two older sisters, who were born with the same disease, both died. “This baby was brought to us when she was two months old,” relates Prof. Reif. “Because of the terrible family history, we immediately did genetic testing.”
Although the health basket committee had discussed approval of this medicine in October, at the time, there were no cases in Israel. Prof. Rief explains that should another case arise, that child will also be able to get coverage for the medicine.
“We thank God for the decision,” said the baby’s father. “We hope our daughter will now be able to grow and develop.”