No longer will Lilike’s parents need to ask for donations for the 700-million-forint (EUR 2 million) treatment.Continue reading
Another six children with spinal muscular atrophy (SMA) will receive state-funded gene therapy, announced the National Health Insurance Fund Management.
“With this, we have become world leaders in public funding for gene therapy treatment,” the institute announced.
Miklós Kásler, Minister of Human Resources, announced earlier this year that a gene therapy drug called Zolgensma for the treatment of SMA disease will become available in Hungary on the basis of individual assessment.
The six individual assessment requests were received and reviewed in turn. The Advisory Committee on Rare Diseases conducted individualized professional examinations and informed parents about the benefits and risks of treatment.
As a result, another six children will receive the Zolgensma gene therapy, which is among Spinraza and Evrysdi therapies, and is one of the most modern personal treatments. Four of them will receive the treatment at Bethesda Children’s Hospital and two at Semmelweis University’s Clinical Center.
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