orphan drugs
| 6 min read
Six studies backed tiratricol's FDA approval for peripheral thyrotoxicosis in MCT8 deficiency, an ultra-rare pediatric thyroid transport disorder
| 3 min read
CGTxchange pairs an AI-enabled evaluation platform with ASGCT's global network to connect deprioritized rare disease programs with new sponsors and investors.
| 3 min read
People with erythropoietic protoporphyria (EPP) must go to great lengths to avoid sun exposure. There have been no approved treatments that address the disease mechanism, but a new drug could change that.
| 10+ min read
Forty years after the Orphan Drug Act passed, researchers advance drug development for neglected rare conditions everywhere from the lab bench to backstage.
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