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Latest news Naples – A 6-month-old girl, suffering from spinal muscular atrophy (SMA), underwent an extremely innovative gene therapy at the Santobono pediatric hospital in Naples. The therapy was authorized in Europe last May and in Italy on November 17. Therefore, it is the first treatment of this type to be carried out in our country. L ‘Type 1 spinal muscular atrophy is a very serious genetic neuromuscular disease, occurs shortly after birth and causes progressive muscle weakness that makes it difficult to breathe and swallow, causing death within two years of life.
Naples, a child saved with the most expensive drug in the world
The drug administered in the Santobono in Naples, considered the most expensive in the world (1.9 million euros for a single treatment), corrects the genetic problem, resulting in complete regression of the disease.
This therapy, in fact, is based on a harmless viral vector that, deprived of its genetic heritage, is capable of carrying the missing human gene to the motor cells of the spinal cord, allowing the production of the missing protein in this disease. The girl has already returned home to her mother and father, after monitoring for a week the side effects associated with the administration of the drug. The rapid use of this drug in Santobono was possible thanks to teamwork that involved the regional pharmaceutical sector, the hospital’s internal services (Acquisition of Goods and Services, Hospital Pharmacy, Business and Health Management) and the entire Neurology team of the Uoc directed by Antonio Varone.
Luigi Russo, father of the girl, thank you:
“Dr. Varone who from the beginning has supported and supports our battle against SMA. At first everything seems black, an endless tunnel. Now, thanks to this drug that arrived before the 6 months of little Sofia, we can all hope and See the expected light at the end of the tunnel. I hope that our little one can guide all the others affected by this disease. Thank you – he concludes – also to all the nurses of the neurology service “.
Anna Maria Minicucci, Extraordinary Curator of the Aorn Santobono Pausilipon, thanked
The Region, the Pharmaceutical Service directed by Ugo Trama and all the nursing and administrative personnel of the company who are committed to achieving this important result of care and innovation for a serious genetic disease. This milestone joins the many achieved in these years by the Santobono Pausilipon Horn of Naples, which has now fully established itself among the most important Italian and European pediatric health realities “.
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