A stem cell transplant effectively eased the signs and symptoms of congenital erythropoietic porphyria (CEP) in a 3-month-old baby girl with severe disease, a case study reported. The results underscore the importance of early diagnosis and treatment to avoid harmful and permanent CEP effects. The case study, “Very…
Givlaari (givosiran) can effectively control symptoms of acute hepatic porphyria (AHP), reducing disease burden, even in patients with low attack rates. That’s according to a post hoc analysis of the Phase 3 ENVISION trial (NCT03338816). As noted by researchers, these results support the long-term benefits of Givlaari…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
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