01669cam a2200301 i 4500 683249073 TxAuBib 20230306120000.0 220728s2023||||||||||||||||||||||||eng|u 9780399576133 hardcover $28.00 0399576134 hardcover $28.00 TxAuBib rda Marcus, Amy Dockser, author. We the scientists : how a daring team of parents and doctors forged a new path for medicine / Amy Dockser Marcus. First edition. New York : Riverhead Books, 2023. xxx, 222 pages ; 22 cm. txt rdacontent n rdamedia nc rdacarrier Includes bibliographical references and index. "Pulitzer Prize-winning reporter Amy Dockser Marcus shows what happened when a group of parents joined forces with doctors and researchers to try to save children's lives. Parents whose children had been diagnosed with the rare and fatal genetic condition Niemann-Pick Type C disease recognized there would never be a treatment in time to save their children if things stayed the same, so the parents set up a collaboration with researchers and doctors in search of a cure. Their social experiment reveals new pathways for treating disease and conducting research"-- Provided by publisher. 20230306. Niemann-Pick diseases Treatment. Rare Diseases Patients. Genetic disorders Patients Family relationships. Parents of children with disabilities.