Zolgensma Treatment for children suffering from spinal muscular atrophy (SMA) is now free, Hungary’s Minister of Human Resources Miklós Kásler announced on Facebook.
According to the minister’s announcement, Lilike will be the first child in Hungary to be treated with the gene-therapeutic Zolgensma treatment, which can dramatically improve their quality of life.
Spinal muscular atrophy is a rare neuromuscular disorder caused by a mutated survival motor neuron 1 (SMN1) gene, which results in the loss of motor neurons and progressive muscle wasting. It can lead to poor head control, difficulty swallowing, scoliosis, and joint contractures.
Zolgensma, marketed by Novartis Gene Therapies, is a therapy which replaces the missing gene with a working copy, essentially bringing it back to working order. Infants who are treated with Zolgensma as soon as possible after their diagnosis are more likely to have better results than those who receive treatment later.
The fact that the Hungarian government will be funding the treatment is excellent news for those parents in Hungary whose children are diagnosed with SMA, since the treatment can cost over 700-million-forint (EUR 2 million).
Parents have previously needed to take to the internet to ask for help in raising money for their children’s treatment, but thankfully this is no longer the case.
Featured photo via Lilike SMA1 Facebook page