With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more
In an ongoing effort to help advocacy groups address unmet needs in rare diseases such as porphyria, therapeutic company Alnylam is calling for applicants for its second annual global ... Read more
The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ investigational candidate givosiran, which will be marketed as Givlaari, for the treatment of adults with acute hepatic porphyria (AHP). The approval comes ... Read more
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more
Genetic variants linked to acute intermittent porphyria vary according to ethnicity, a systematic analysis among eight ethnic populations shows. The finding adds a new layer of knowledge that may ... Read more
Most acute hepatic porphyria (AHP) patients with recurrent attacks also experience significant chronic symptoms between attacks, with more than 75% of them needing to be hospitalized or treated with hemin, ... Read more
Adult patients with erythropoietic protoporphyria (EPP) who live in the United States can now be treated with Scenesse (afamelanotide) to increase their tolerance to sunlight. Scenesse has been approved ... Read more
Toxic accumulation of protoporphyrin IX in the skin and liver of people with erythropoietic protoporphyria (EPP) — the most common porphyria of childhood — occurs through the action of the ... Read more
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more
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