Six More Children With SMA to Receive Free Gene Therapy

Csongor Szij 2021.06.17.

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.

Fact

SMA is the abbreviation for spinal muscular atrophy due to a genetic defect in the SMN1 gene. The SMN1 gene encodes SMN, a protein necessary for the survival of motor neurons. Without proper medication, the loss of these neurons leads to the atrophy of limbs, then respiratory problems. There are two medications for this illness. One is really painful and requires constant meetings with doctors, physiotherapy, and lots of exercises at home so muscles can function. The other is a once-in-a-lifetime treatment, and it helps muscles function without further intervention. This medication, called ZOLGENSMA, could deposit a new, working copy of the SMN1 gene for renewed muscle function. Its price is HUF 730 million (EUR 2 million), an unaffordable amount of money for most households as it equals the average salary of more than 200 years.

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.

State Funds SMA-Afflicted Child's Treatment for First Time

No longer will Lilike’s parents need to ask for donations for the 700-million-forint (EUR 2 million) treatment.Continue reading

Featured photo illustration by Rene Asmussen/pexels.com