Disc Medicine has entered into an exclusive worldwide licensing agreement to develop and commercialize Roche’s bitopertin, an oral…
Articles by Patricia Inácio, PhD
Three women in Nepal were diagnosed with the rarest type of porphyria, called congenital erythropoietic porphyria or CEP,…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
Rapid and severe damage to peripheral nerves, those sending sensory and motor information from the brain and spinal cord to…
People with acute intermittent porphyria (AIP) have structural changes in the brain, namely an enlargement of the brain’s ventricles…
A long-term study seeking to deepen the understanding of the natural history of porphyrias — both acute, typically affecting…
Real-world data support the effectiveness of Scenesse (afamelanotide) in extending tolerance to sunlight among adults with erythropoietic protoporphyria…
People with acute intermittent porphyria (AIP) who were treated with Alnylam Pharmaceuticals’ RNA-based therapy Givlaari…