DNA double helix graphic

Clinical trials for people with cancer and rare diseases

Sometimes, just one small change on one small part of a gene can cause or contribute to a debilitating malfunction that affects the whole human body. Identifying these specific – and often rare – mutations is important, because drugs can then be researched and developed to target them. And that’s what researchers at Blueprint Medicines are working hard to do – right at this second, in fact. You see, we believe that just because a disease is rare, that doesn’t mean it should be neglected.