- It fills us with great pride that we are on the European podium in the care of patients with spinal muscular atrophy," said Prof. Maria Mazurkiewicz-Bełdzińska of the Medical University of Gdansk during a panel on rare diseases during Priorities and challenges in Polish and European drug policy 2024. However, this does not mean that there are no challenges in this matter. One of them is the expansion of indications for therapies, especially gene therapy, in the context of patients with four copies of the SMN2 gene.
- Diagnosis and therapy are two key elements in the care of patients with rare diseases. Scientific advances are making it possible to diagnose patients very effectively and to look for points of entry for specific therapy. This has worked wonderfully in the case of gene therapy for children with SMA," said Deputy Health Minister Ursula Demkow.
The starting point for gene therapy is newborn screening. At the end of March 2022, all Polish newborns are covered. The introduction of screening has made it possible to diagnose the disease in the m...
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