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September 20, 2019September 20, 2019

TB Antibiotic Shows Promise in Mice with Erythropoietic Protoporphyria, but Researchers say More Human Studies are Needed

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Isoniazid, an antibiotic to treat tuberculosis, produces promising results in mice with erythropoietic protoporphyria (EPP), but the standard dose is not high enough to have an effect in humans, ... Read more

September 13, 2019September 13, 2019

Givosiran Continues to Show Promise in Treating AHP, Trial Data Shows

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Alnylam Pharmaceuticals announced new findings from several clinical trials that continue to demonstrate the effectiveness of givosiran, the company’s investigational therapy for the treatment of acute hepatic porphyria (AHP). ... Read more

September 6, 2019September 6, 2019

Glycyrrhizin May Be Effective in Patients with Alcohol-linked PCT, Case Report Says

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Glycyrrhizin may be an effective treatment option for patients who develop porphyria cutanea tarda associated with alcohol abuse, a case report says. The case report study, “A first report of ... Read more

August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 30, 2019August 30, 2019

PCT Linked to Higher Risks of Gastrointestinal Disease, Cancer, and Premature Death, Study Suggests

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People with porphyria cutanea tarda (PCT) have higher risks of developing gastrointestinal diseases and certain types of cancer, compared with individuals from the general population, a study says. They ... Read more

August 29, 2019August 29, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 22, 2019August 22, 2019

Alnylam Partners with Ironwood to Make Doctors More Aware of Givosiran’s Potential

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Alnylam Pharmaceuticals and Ironwood Pharmaceuticals are joining forces to better inform healthcare professionals about givosiran, Alnylam’s potential therapeutic for acute hepatic porphyria (AHP). Givosiran (also known as ALN-AS1) is an RNA-based ... Read more

August 20, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

August 9, 2019September 13, 2019

FDA Grants Priority Review to Givosiran for Treating Acute Hepatic Porphyria

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The U.S. Food and Drug Administration (FDA) has accepted and granted priority review status to Alnylam Pharmaceutical’s new drug application (NDA) seeking approval of givosiran for treating acute hepatic porphyria (AHP). Priority review means the FDA’s decision ... Read more

August 2, 2019August 2, 2019

New Mutation in FECH Gene Linked to EPP, Study Shows

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Erythropoietic protoporphyria (EPP) with mild or no liver disease can be caused by two co-occurring mutations in the FECH gene, researchers report. One of the two mutations has not ... Read more

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