Gene therapy for rare form of blindness wins US approval
U.S. health officials on Tuesday approved the nation’s first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major advance for the emerging field of genetic medicine.
The approval for Spark Therapeutics offers a life-changing intervention for a small group of patients with a vision-destroying genetic mutation and hope for many more people with other inherited diseases. The drugmaker said it will not disclose the price until next month, delaying debate about the affordability of a treatment that analysts predict will be priced around $1 million.
The injection, called Luxturna, is the first gene therapy approved by the Food and Drug Administration in which a corrective gene is given directly to patients. The gene mutation interferes with the production of an enzyme needed for normal vision.
http://abcnews.go.com/Health/wireStory/gene-therapy-rare-form-blindness-wins-us-approval-51884772
ABD dünyanın en pahalı ilacını tartışıyor… Spark Therapeutics isimli bir firma genetik olarak doğuştan kör hastalar için uygulayacağı gen tedavisi için resmi onayı aldı. 1 milyon dolarlık ilacın kullanılacağı tedaviyi ekonomik olarak değerli kılan şey ise dünyanın en pahalı ilaçları arasında yer alacak olması. İlaç doğuştan kör insanların çok parlak cisimleri görmesini sağlıyor.
