The U.S. Food and Drug Administration (FDA) has rejected an application from Disc Medicine seeking accelerated approval of its experimental oral therapy bitopertin as a treatment for erythropoietic protoporphyria (EPP). According to Disc, the FDA said available clinical data aren’t sufficient to conclude that bitopertin is likely to…
A study in mice suggests that genetic makeup and diet may influence a person’s susceptibility to developing sporadic porphyria cutanea tarda (PCT), the most common form of porphyria, after exposure to environmental triggers. When researchers exposed mice to excess iron, a known trigger of PCT, only some developed…
A woman with acute intermittent porphyria (AIP) developed lasting nerve damage after repeated disease attacks, resulting in distinctive hand changes known as “claw hands,” according to a case report from Taiwan. In this condition, the fingers curve into a claw-like position due to damage affecting the nerves that control…
Discussion
Shortly after I was diagnosed with acute intermittent porphyria (AIP) in October 2020, I discovered the American Porphyria Foundation’s website, which is dedicated to supporting patients with porphyria. The site offers valuable information about the condition and its approved treatments, as well as a store featuring…
Becoming educated and informed about porphyria is a good first step. Here you’ll find general information about the disease, including the types, diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of some of our columnist’s words of wisdom to help you along on your porphyria journey.
There is no cure for porphyria yet, but there are therapies that can help manage the disease. Here you can find more information about some of the approved treatments for porphyria.
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