For Rare Disease Patients, A Pathway To Hundreds Of New Therapies


For Rare Disease Patients, A Pathway To Hundreds Of New Therapies

Many rare disease patients use drugs off-label based on limited data because they have no better options available. In fact, it is estimated that 95 percent of rare diseases do not yet have a single FDA-approved treatment. One of the authors of this article, David Fajgenbaum, is a physician-scientist and a patient with a rare and incurable condition called idiopathic multicentric Castleman disease (iMCD). An off-label treatment saved his life one day after he was administered his last rites in 2010. He has had multiple relapses and received 11 off-label treatments with varying effectiveness. He is currently on an off-label treatment that he …

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