Signalise: a Dazzle4Rare Podcast

EP39 Rare and Relevant News Stories of 2023 TL;DR and Update

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Episode notes

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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