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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Don't miss an episode of Signalise! Be sure to ✔️FOLLOW or ✔️SUBSCRIBE via your podcast app of choice. You can also follow #Signalise news on @Dazzle4Rare on Facebook , Instagram , and Twitter. You can find us on LinkedIn as well at Dazzle4Rare.

A quick "bonus" episode to drop to let you know that we'll be making some scheduling changes and 2024 will hopefully fingers crossed be a year of positive changes. But first, a big thanks to our faithful listeners for tuning in today and to this bi-monthly podcast. From this month, January 2024, we're shifting our podcast schedule to Fridays, keeping the podcast bi-monthly.

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Don't miss an episode of Signalise! Be sure to ✔️FOLLOW or ✔️SUBSCRIBE via your podcast app of choice. You can also follow #Signalise news on @Dazzle4Rare on Facebook , Instagram , and Twitter. You can find host us on LinkedIn as well at Dazzle4Rare.

In this festive episode, Kimberly shares a little holiday spirit by with heart-warming stories of children with rare conditions and their families finding hope. She also shares International Universal Health Coverage Day brought to our community's attention by Dr. Eleonora Passeri of Rare Special Powers (IT). 1. International Universal Health Coverage Day: https://www.un.org/en/observances/universal-health-coverage-day 2. Emma's Dravet Syndrome Story:

https://www.cbsnews.com/philadelphia/news/dravet-syndrome-rare-disease-emma-watson

3. George's Lung Condition: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1403838

4. George's Christmas Celebration:

https://www.coventrytelegraph.net/news/real-life/rugby-boys-first-christmas-without-28203691

5. Poppy's Musical Tribute: https://www.thetelegraphandargus.co.uk/news/23958762.bradford-teen-re-releases-song-memory-young-relatives

6. Buy a Copy of the Song: https://beyondrecords.uk/

7. Cure 4 The Kids' Challenge:

https://nevadabusiness.com/2023/11/celebrities-come-together-to-support-the-happy-xmas-shout-out-challenge

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Don't miss an episode of Signalise! Be sure to ✔️FOLLOW or ✔️SUBSCRIBE via your podcast app of choice. You can also follow #Signalise news on @Dazzle4Rare on Facebook , Instagram , and Twitter. You can find host us on LinkedIn as well at Dazzle4Rare.