Prognosis
Some Patients’ Best Hope for a Cure Is to Develop It Themselves
Ultra-rare diseases aren’t profitable for drug makers, so many sufferers go it alone
Helen Evangelista and her son Matthew at St. Mary's Children's Hospital in New York, on Aug. 13.
Photographer: Christopher Occhicone/BloombergThis article is for subscribers only.
It was a life-or-death situation for young Matthew Evangelista. His mother, Helen, had nowhere to turn.
Doctors couldn’t help. Matthew was born with a disease that affects only 200 people in the world, and there was no treatment. With such a small number of patients, pharmaceutical companies saw no profit in developing a drug therapy to fight it.