Feb. 28, 2026

Rare Diseases

Show Notes
Transcript

Question 1: Rare Disease Day was designed to symbolize scarcity by landing on the calendar’s rarest date—so in leap years, on what date is it observed?

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?

You may also like ODD ONE OUT. a new podcast with Mark Ellison! Mark gives you 4 words, you decide which word does not belong. Three rounds daily, quick and fun,

Portions of today's trivia podcast were made with the help of AI.

WEBVTT

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Calarrogus shark media. Hi. I'm Mark Ellison and today's theme

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is Rare Disease Day, Medical Breakthroughs, healthcare heroes and scientific advances.

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Question one. Rare Disease Day was designed to symbolize scarcity

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by landing on the calendar's rarest date. So in leap

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years on what date is it observed? Question two? Which

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landmark US law passed in nineteen eighty three transformed rare

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disease treatment development by offering incentives like seven years of

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market exclusivity, tax credits, and fee waivers to drug makers.

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Question three. In twenty nineteen, the FDA approved a one

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time gene therapy that delivers a working SMN one gene

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to infants with a devastating neuromuscular disorder. Which rare disease

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does this treatment target? Question four. Cystic fibrosis care was

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revolutionized in twenty nineteen by a three medicine CFTR modulator

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that dramatically improved lung function for many patients. What is

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the brand name of this breakthrough combination therapy? Question five?

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Which patient advocate founded the National Organization for Rare Disorders

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NORD and played a pivotal role in passing the Orphan

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Drug Act, giving a voice to families facing rare conditions.

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We'll be right back with these answers after this break.

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Welcome back. Question one, on what date is Rare Disease

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Day observed in leap years? Answer February twenty ninth. Launched

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by EUROTUS in two thousand and eight, Rare Disease Day

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uses February twenty ninth to highlight the rarity of these conditions.

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In non leap years. It's observed on February twenty eight.

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The day raises global awareness for the roughly three hundred

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million people living with rare diseases. Question two What nineteen

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eighty three US law spurred rare disease drug development? Answer?

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The Orphan Drug Act. The Orphan Drug Act jump started

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innovation for conditions affecting fewer than two hundred thousand Americans.

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With advocacy from NORD and others, it helped lead to

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hundreds of approved therapies for previously neglected diseases. Question three.

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Which disease is treated by the gene therapy ZOLGENZMA. Answer

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spinal muscular atrophy SMA on asemogene abaparvavec ZOLGENZMA uses an

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AAV nine vector to provide a functional SMN one gene,

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helping preserve motor neurons. It marked a major breakthrough for SMA,

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a leading genetic cause of infant mortality. Question four, What

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is the name of the triple CFTR modulator approved in

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twenty nineteen? Answer trikafta trikafta alexicaft slash tesacaft slash ivocaft

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targets the defective CFTR protein, especially patients with the common

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F five hundred eight Dell mutation. It has significantly improved

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quality of life and outcomes for many with this rare

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genetic disease. Question five. Who founded NORD and champion the

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Orphan Drug Act? Answer? Abbi Myers. Abby Myers mobilized patients

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and families to push for policy change, leading to the

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Orphan Drug Act's passage. NORD continues to support research, awareness,

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and patient services across thousands of rare diseases. That concludes

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today's five daily Trivia questions. I'm Mark Ellison, and today's

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questions were crafted with the help of AI technology. See

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you tomorrow.

Rare Diseases

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