Articolo speciale
Malattie rare a cavallo dell’innovazione: una sfida etica e scientifica
Rare disease and recent advances in medicine: an ethical and scientific challenge
Alberto Tommasini, Andrea Magnolato, Serena Pastore, Irene Bruno
Clinica Pediatrica, IRCCS Materno-Infantile “Burlo Garofolo”, Trieste
Dicembre 2018 - pagg. 628 -634
Abstract
Recent advances in medicine are providing new opportunities to treat rare and complex
disorders. Precision therapies are being developed to target molecular processes crucial
to the disease pathogenesis. Media often present technological advances raising great
hopes. However, the journey on the road of medical innovation can be extremely
bumpy for some patients. Indeed, subjects living during a period of innovation and medical
changes may just face failures, both in terms of frustrated hopes as well as suffering.
Indeed, novel treatments do not always lead to a cure for the disease and the choice
to join a clinical trial with innovative medications can be hardly challenging, both as
concerns science and humanism. The paper describes four stories of children with rare
disorders who had different outcomes and discusses how difficult the best scientific and
ethical choices can be when novel treatments are proposed
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Corrispondenza: alberto.tommasini@burlo.trieste.it