Saturday , June 19 2021

6 month old girl rescued in Naples with the most expensive drug in the world






The little girl, suffering from spinal muscle atrophy, is treated at the Santobono Hospital: the disease has subsided and the girl has returned home.


Type 1 spinal muscular atrophy is a very serious genetic neuromuscular disease, which occurs immediately after birth and causes progressive muscle weakness that interferes with breathing and swallowing, leading to death within 2 years. On the other hand, the gene therapy given to Santobono allows complete recovery: it is based on a viral vector that is considered harmless and is used as a “vector” to carry the missing human genes in motor cells in the spinal cord,
which is thus able to produce the missing protein.

The little girl, after treatment, was watched for a week for side effects, and then she was able to go home with her parents. Who thanks the doctors for “supporting them in our battle against high school. At first everything looked black … the tunnels without end … Now thanks to this drug arrived.
before six months little Sofia we can all hope and see the long awaited light at the end of the tunnel. I hope our little girl can guide everyone with this disease. Thanks also to all the nurses in the neurology department “.

As a result, Dr. Antonio Varone underscores, team director of the Neurology Operations Unit, made possible thanks to “the introduction of innovative therapies that have contributed to radically changing the clinical history of the disease, which remains one of the main causes today. Infant mortality The emergence of such therapeutic solutions creates a need for greater awareness. will early diagnosis become possible through implementation of neonatal screening projects becoming more relevant than ever.




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