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January 17, 2020January 17, 2020

Non-Gastrointestinal Symptoms in Crohn’s Patients Could Signal Acute Intermittent Porphyria, Case Report Suggests

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Abnormal non-gastrointestinal symptoms in patients with Crohn’s disease may indicate the presence of acute intermittent porphyria, a case report of a 23-year-old patient shows. The study, “Acute Intermittent Porphyria ... Read more

January 10, 2020January 10, 2020

Iron-Regulating Mechanism May Open Door to New Porphyria Therapies, Study Suggests

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A tissue-specific mechanism was found to take part in the regulation of iron levels in the body, a discovery that may have implications for new therapies for diseases such ... Read more

January 3, 2020January 3, 2020

Porphyria Diagnosed After PRES Suspected in Teenager

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In an unusual case, a 16-year-old girl in India was diagnosed with acute intermittent porphyria (AIP) after radiological findings were suggestive of posterior reversible encephalopathy syndrome (PRES). The combination of abdominal ... Read more

December 20, 2019December 20, 2019

Alnylam Selects PANTHERx to Distribute its Recently Approved AHP Treatment Givlaari

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Alnylam Pharmaceuticals has tapped PANTHERx Rare Pharmacy to be a limited distribution partner for its newly approved therapy Givlaari (givosiran) to treat acute hepatic porphyria (AHP). The medication was ... Read more

December 10, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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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

December 6, 2019December 6, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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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

December 6, 2019December 17, 2019

Alnylam Calls for Applicants for ‘Advocacy for Impact’ Grants Helping Rare Disease Communities

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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

November 22, 2019November 26, 2019

Givosiran Approved by FDA for Treating Adults with Acute Hepatic Porphyria

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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

November 15, 2019November 16, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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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

November 8, 2019November 8, 2019

HMBS Mutations Linked to Acute Intermittent Porphyria May Vary According to Ethnicity, Study Suggests

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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

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