New Muscular Dystrophy Drug Granted FDA Orphan Designation in US
So, what is the drug and how does it work?
The drug, called BB-301 is considered to be a kind of gene therapy designed to target the specific mutated gene that causes OPMD. So, the drug will “silence” the defective gene that has been present since birth, and replace it with a copy of a healthy gene and as a result, minimizing the symptoms of the disorder.
Referred to as ‘silence and replace’ technology this is truly a groundbreaking drug and the technology will hopefully lead to this kind of gene therapy being used to help people with other kinds of orphan disease in the future.