A therapy is granted Orphan Drug designation if the FDA believes that this therapy will treat, prevent, or diagnose rare conditions. Rare conditions, in the United States, are those affecting…
EDIT-301 is an investigational, gene editing medicine developed by Editas Medicine for the treatment of beta thalassemia. As recently reported in the Business Insider, EDIT-301 has just been granted the…
Rare Pediatric Disease designation is granted by the FDA to drugs or biologics intended to treat life-threatening diseases affecting individuals aged 18 or younger. In this case, rare conditions are…
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