Dying Patients With Rare Diseases Struggle to Get Experimental Therapies
When patients with common terminal illnesses such as cancer seek permission for compassionate use of therapies in the testing stage, their requests often are approved. But those with more unusual illnesses say drug companies are rarely willing to provide access.
Gene Screenings Hold Disease Clues, but Unexplained Anomalies Often Raise Fears
Multiple-gene panel tests are frequently offered to patients at risk for diseases such as cancer that can assess more than 80 genes. But in screening a wide variety of genes, doctors might see a variant that hasn’t yet been deciphered and be unable to explain its significance, leaving patients with concerns and no answers.
