For the first time, researchers at Boston Children’s Hospital have created a drug for one person. In 2016, a six-year-old patient was diagnosed with Batten’s disease, a neurological problem without cure.
An analysis of the DNA revealed that the cause was the mutation of a gene called Cln7. Once the defect was isolated, the researchers devised a small DNA fragment that could “mask” the defect.
Once tested on animals the drug, which was called “milasen”, was infused into the baby, after the FDA approval for the test, with positive results.