Patients are very satisfied with Givlaari (givosiran), the only approved treatment that targets the cause of acute hepatic porphyria (AHP) to help reduce attacks, according to a new study based on interviews with more than 20 people with AHP in the U.S. and Europe. Indeed, these AHP patients —…
John Manak, PhD, a professor and human genetics disease expert at the University of Iowa, emailed me to ask if I’d speak to his class about living with acute intermittent porphyria (AIP). The class was called “Good Genes Gone Bad.” Little did I know at the time how much…
Women with acute hepatic porphyria (AHP) who experience sporadic attacks reported facing chronic symptoms and significant disruptions to their daily activities around each attack, according to a recent study. During patient interviews, these women also frequently described symptom “flares” that they did not consider to be an acute porphyria…
My 19-year journey to an acute hepatic porphyria (AHP) diagnosis was fraught with misdiagnoses and traumatic misunderstandings. Sadly, it is not a unique story within the rare community. This column is dedicated to those with mysterious symptoms, who are in the thick of searching for answers. Don’t…
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…
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