Rare diseases: Fixing fate

Print section UK Only Article:  standard article Issue:  The new political divide Fly Title:  Rare diseases Location:  MILAN Main image:  20160730_wbp503.jpg Rubric:  New medical cures may mean changes in drugmakers’ business models WHEN families leave the genetic institute at the San Raffaele Hospital in Milan, they are still anxious. Later, many will come to see the day their children received gene therapy as a blessed new start. Youngsters who had been sentenced to short lives, full of suffering caused by faulty DNA, get better and thrive. Cures for rare genetic diseases, both for children and adults, were once no more than a dream, but now they are set to become commercial reality. Gene therapies take sections of correct DNA and insert them into cells, often using viruses. Once inside the cell, the new DNA produces the protein that was formerly missing and the fault is fixed. Last week America’s Food and Drug Administration ...
Source: Biotechnology - Category: Biotechnology Source Type: news