Poland will refund gene treatment of spinal muscular atrophy (SMA) in newborns and infants up to the age of 6 months. Zolgensma - dubbed the most expensive medicine in the world - is a chance for a normal life for babies affected by this rare genetic disease, which not so long ago was considered incurable. Each year approx 40-50 children with SMA are born in Poland.
Poland's Health Minister Adam Niedzielski announced on Monday (August 22) that the most expensive single-dose-only medicine for treating spinal muscular atrophy (SMA). "I'm talking about Zolgensma, which will be dedicated to babies aged up to 6 months. Of course it will be applied after carrying out a screening programme," the minister said on Monday. "We are also introducing a drug that will supplement treatment with nusinersen," he added.
"As part of the comprehensive programme of fight against SMA we are also introducing a medicine which will complement treatment with nusinersen, which due to difficult administration method sometimes requires switching to other forms of administration," Niedzielski said.
Nusinersen is used to treat spinal muscular atrophy associated with a mutation in the SMN1 gene. It is administered directly to the central nervous system using intrathecal injection.
Head of the Department of Neurology at the Medical University of Warsaw Prof. Anna Kostera-Puszczyk said that thanks to refunding of the SMA gene therapy, Poland would join countries which guarantee comprehesive care for SMA patients.
Apart from refunding two therapies for this rare genetic disease, in late March Poland has also launched an SMA screening tests programme available in all regions.
Thanks to screening tests, 39 newborns have been qualified for treatment early treatment, which is a chance for them to have a normal life and avoid paralysis or difficulties with walking.
The genetic root cause of SMA is the SMN1 gene that is missing or not working properly. When this main gene is missing or not working properly, the body cannot make enough survival motor neuron (SMN) protein, which is needed for motor neuron cell survival.
Everyone is born with a certain amount of motor neuron cells, which are responsible for communicating with the arms, legs, throat, and many other areas in the body to tell them to work properly. Without enough SMN protein, select motor neuron cells throughout the body may lose function and die. As a result, patients with SMA experience muscle weakness and may develop difficulty breathing, swallowing, or speaking.
Źródło: TVN24 News in English, tvn24.pl, PAP
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