Disc Medicine’s experimental oral therapy bitopertin significantly reduced levels of protoporphyrin IX (PPIX), a light-sensitive molecule, and significantly increased pain-free time in light in a Phase 3 trial involving people with erythropoietic protoporphyria (EPP). That’s according to top-line data from the global Phase 3 APOLLO trial (NCT06910358),…
Discussion
What would you do in a zombie apocalypse? The answers might vary, but they usually look something like this: Stock up on food and water. Gather family and friends. Find shelter. Figure out how to defend yourself. But when you have a chronic or rare illness like porphyria…
Discussion
A woman with acute intermittent porphyria (AIP) who suffered an unusually severe attack marked by extensive muscle breakdown, kidney injury, profound weakness in all four limbs, and respiratory failure was helped by long-term treatment, according to a case report from Croatia. Treatment with hemin followed by long-term Givlaari…
Discussion
Preventive treatment with intravenous hemin — a therapy commonly used to treat acute porphyria attacks — reduced attack risk over the following week in people with acute hepatic porphyria who received their scheduled preventive dose rather than placebo, according to results from a small clinical trial. “This randomized, double-blinded, placebo-controlled…
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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