“Today we presented the extension of gene therapy, which had previously been a real revolution for children affected by the most serious form of the disease (SMA 1). Today, having the possibility of therapy for children also for form 2 is truly an indescribable joy, children who, although with a less serious form of the disease, still experience a progressive disability”. Thus Anita Pallara, president of the SMA Families Association, comments on the extension of the gene therapy onasemnogene abeparvovec (Zolgensma*) also to SMA type 2, in a meeting with the press organized today in Rome by Novartis.
“We remember that SMA is a progressive neuromuscular disease – explains Pallara – therefore a child with SMA, even with type 2 SMA, will develop an increasingly serious disease over the course of his life. Therefore having the possibility of receiving therapy, which has a 'one shot' administration, i.e. only once in a lifetime, making it free even from repeated hospital admissions, is a huge revolution and responds to the ever-present and reiterated need of our community to receive therapies that are as effective and safe as possible and that can lead to a radical change in the course of the pathology”.
In addition to gene therapy, continues the president of Famiglie Sma, “one of our main objectives” is the extension of “neonatal screening, because there is a Lea decree and an update and we are waiting, we have the scientific data to support us they say how important it is to treat children before the symptoms appear. Having regions where this is possible and others where unfortunately it is not yet possible is an inequality in terms of health that goes against our constitutional principles. We have the therapies, we have the tests. we have everything, we need newborn screening to be extended across the entire territory”, he concludes.
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