Rare Diseases
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Rare Diseases
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About this listen
Question 2: Which landmark U.S. law, passed in 1983, transformed rare-disease treatment development by offering incentives like seven years of market exclusivity, tax credits, and fee waivers to drug makers?
Question 3: In 2019, the FDA approved a one-time gene therapy that delivers a working SMN1 gene to infants with a devastating neuromuscular disorder; which rare disease does this treatment target?
Question 4: Cystic fibrosis care was revolutionized in 2019 by a three-medicine CFTR modulator that dramatically improved lung function for many patients; what is the brand name of this breakthrough combination therapy?
Question 5: Which patient advocate founded the National Organization for Rare Disorders (NORD) and played a pivotal role in passing the Orphan Drug Act, giving a voice to families facing rare conditions?
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Portions of today's trivia podcast were made with the help of AI.
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