Hats off to these pioneers for their work to improve the lives of their patient community!!!
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- In honor of Rare Disease Day, February 28th, 2023, Please Support This Film
- We have a lot planned for 2023, and are excited to have you on this journey with us.
- Two Leading Rare Disease Organizations Join Forces to Enable and Accelerate Patient-Led Engagement and Advance Research
- Menkes Disease Treatment on the Horizon, After Nearly Three Decades
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