Children's Wisconsin to offer new gene therapy for Sanfilippo Type A
Children's Wisconsin to offer new gene therapy for Sanfilippo Type A
A breakthrough treatment for a rare childhood genetic disorder is giving new hope to families. The FDA recently approved the gene therapy that will be available in Southeast Wisconsin.
MILWAUKEE - A breakthrough treatment for a rare childhood genetic disorder is giving new hope to families. The FDA recently approved the gene therapy that will be available in Southeast Wisconsin.
FAYUVI gene therapy
What To Know:
The gene therapy is called FAYUVI and is used to treat children with Sanfilippo Type A. The one-time dose is allowing patients' families to envision a healthy future for their kids.
"There is no cure. There is no treatment. Now we have a treatment," Roger Benda said.
Six months ago, his 4-year-old son Ozzie was diagnosed with Sanfilippo type A. It is like Alzheimer's disease in kids. It is a rare genetic disorder where children’s bodies are unable to break down certain sugar molecules. Over time, buildup damages the brain and is ultimately fatal.
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"We had a lot of sadness and grief obviously," Benda said.
Days ago, the FDA approved the first-ever treatment for the disease. Children's Wisconsin was named one of the hospitals able to administer therapy.
Children's Wisconsin
"Prior to this gene therapy, we haven’t had any treatments available for this specifically," said Dr. Michael Finkel of Children’s Wisconsin.
FAYUVI is a one-time dose that provides a functional copy of the gene that is not working. It is not a cure. It is shown to preserve neurologic function.
"Now we can actually provide a treatment that alters the entire natural history of this disorder," Finkel said.
This Illinois family does not know if, when or where they might be able to get the treatment. But they are hopeful about the breakthrough.
"We can enjoy the time we have with him and hopefully don’t have to watch him decline as quickly," Benda said.
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Children's Wisconsin is one of 20 qualified treatment centers administering the therapy in the U.S., and one of four in the Midwest. The Benda family said they are already talking with their insurance about the treatment. It could be months before Ozzie gets approval for it.
Portions of this article were formatted using A.I. FOX6’s Ashley Sears and an editor reviewed it for accuracy and tone prior to publishing.
The Source: Information in this story was gathered from Roger Benda, Children's Wisconsin, Dr. Michael Finkel and FOX6 News reporting.