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Welcome to the first 2024 episode of Signalise: a Dazzle4Rare podcast! Remember, we’re now airing on Fridays. In this episode, we’re looking at your rare and relevant TL;DR for 2023 with some of the rare disease stories and news that had the most eyes on in 2023.
Welcome to the first 2024 episode of Signalise: a Dazzle4Rare podcast! Remember, we're now airing on Fridays. In this episode, we reflect on 2023's significant rare disease developments and look forward to more progress in 2024.
Most Read Rare Disease News of 2023
- FDA's first gene therapy approval for DMD.
- Promising treatments for idiopathic pulmonary fibrosis and hemolytic disease of the fetus and newborn.
- Risks of colitis in MS patients using ocrelizumab and rituximab.
Sources:
AJMC's Top 5 Rare Disease Articles of 2023
https://www.ajmc.com/view/top-5-most-read-rare-disease-articles-of-2023
The 10 Biggest Stories in Rare Disease for 2023
https://www.rarediseaseadvisor.com/features/the-10-biggest-stories-in-rare-disease-for-2023/#:~:text=The%20US%20Food%20and%20Drug,and%20still%20able%20to%20walk
New York Times - Family in ‘Take Care of Maya’ Documentary
https://www.nytimes.com/2023/11/10/us/take-care-of-maya-trial-damages-kowalski.htmlResearching Reform - What Happened to Maya
https://researchingreform.net/2022/10/20/what-happened-to-maya-when-professional-arrogance-and-ignorance-collide/
Family in ‘Take Care of Maya’ Documentary Is Awarded $261 Million
https://www.nytimes.com/2023/11/10/us/take-care-of-maya-trial-damages-kowalski.html
What did Gypsy Rose Blanchard do? What to know about the case as she is released from jail
https://www.nbcchicago.com/news/local/what-did-gypsy-rose-blanchard-do-what-to-know-about-the-case-as-she-is-released-from-jail/3315071/
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The Dazzle4Rare event and Signalise podcast amplify the voices of rare disease and associated communities by sharing their stories, new, events, and more. Working together, we have strength in numbers, amplifying our critical messages.
We feature guests and discuss relevant topics for rare disease patients, caregivers, and those in the URCIID community.