When his infant son was diagnosed with aย rare disease, a Canadian father was dismayed to discover there was no treatment or cure. So he set out to make one himself. There is no treatment currently approved by the U.S. Food and Drug Administration (FDA) for SPG50.
After the shock of the diagnosis, [Terry] Pirovolakis immediately started researching, with a focus on finding aย gene therapyย that could help his son.
The disease typically has a life expectancy of 20 to 30 years if left untreated.
It costs about $1 million to make the drug for each child, Pirovolakis said, and another $300,000 or soย to treat the patient in the U.S. at the hospital….
Without the backing ofย major drug companies, however, there isnโt funding available to get the therapies to the children who need them….
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“The treatment is here, just literally sitting in a refrigerator, ready to go,” Lockard said. “Doctors are ready. There just isn’t enough money to make it happen.”
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Ideally, after the drug is approved โ which could take three to five years, Pirovolakis estimates โ SPG50 will be added to hospitalsโย newborn screening programsย and every child with the disease will be able to get the therapy.
