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You suffer from spinal muscular atrophy (SMA). The therapy corrects the genetic problem, 1.9 million euros for a single treatment
At the Santobono pediatric hospital in Naples, a girl under six months of age with spinal muscular atrophy (SMA) underwent an extremely innovative gene therapy – according to hospital sources – authorized in Europe last May and in Italy on May 17. November. It is – refer to Santobono – the first treatment of this type to be carried out in our country. Type 1 spinal muscular atrophy is a very serious genetic neuromuscular disease that occurs shortly after birth and causes progressive muscle weakness that makes breathing and swallowing difficult, leading to death within 2 years.
The drug administered to 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 viral vector that is rendered harmless and is used as a shuttle to transport the missing human gene in the motor cells of the spinal cord, allowing the production of the missing protein in this disease.
Anna Maria Minicucci, extraordinary commissioner of the company Santobono Pausilipon says “I thank the Region, the Pharmaceutical Service directed by Ugo Trama and all the nursing and administrative staff 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 recent years by Orn Santobono Pausilipon in Naples, which has now fully established itself among the most important Italian and European pediatric health realities “.
The President of the Campania Region, Vincenzo De Luca, declares: “Another extraordinary demonstration of excellence in health care in Campania. An even more significant teamwork, considering the age of the child and the possibility that the Santobono de poder offers first time in Italy, an avant-garde therapy that caused the resignation of the little patient. I would like to thank the strategic management, the doctors and all the Santobono staff for this great result obtained “.
The girl has already returned home. Returning home to parents after being monitored for a week for side effects related to the administration of the medication. The rapid use of this drug in Santobono was possible, it stands out, “thanks to an exceptional teamwork that involved the regional pharmaceutical sector, the internal services of the hospital: Acquisition of Goods and Services, Hospital Pharmacy, Business Management and Health and all the team of the Neurology Unit headed by Dr. Antonio Varone. “” In recent years, the introduction of innovative therapies – points out Varone – has contributed to radically change the clinical history of the disease, which today remains one of the main causes of infant mortality. The advent of these therapeutic solutions makes the need to increase awareness about early diagnosis that can be achieved by implementing neonatal screening projects. ”
Luigi, the little girl’s father, adds: “I thank Dr. Varone, who from the beginning supported and supports our battle against SMA. At first everything seems black … An endless tunnel … Now thanks to this drug Arriving before six months of little Sofía we can all wait and see the long-awaited light at the end of the tunnel. I hope that our little girl can guide all the others affected by this disease. Thanks also to all the nurses in the neurology department “.
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