Editor’s Choice: Developments In Beta Thalassemia, Eosinophilic Esophagitis and Congenital Adrenal Hyperplasia

Happy Friday!

This week, we have details on an investigational compound for beta thalassemia. Also, we break down the details of a new study that examines psychiatric illness rates among eosinophilic esophagitis patients. And, a spotlight on CAH explores why rare clinical trials are essential to uncovering potential patient solutions.


FDA Grants Rare Pediatric Disease Designation for Experimental Beta Thalassemia Drug

EDIT-301 is an investigational, gene editing medicine developed by Editas Medicine for the treatment of beta thalassemia.




Study of the Week: Eosinophilic Esophagitis Appears to Increase Rates of Psychiatric Illness

The increased risk was also still present even after accounting for other conditions, such as asthma, celiac disease, and inflammatory bowel disease.




Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)

Without patient volunteers, groundbreaking research and potential solutions can’t move forward.




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