FDA Approved: Cuvrior for Wilson Disease
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FDA Approved: Cuvrior for Wilson Disease

For the last 52 years, individuals with Wilson disease have been relying, largely, on the same treatment options. These options, designed to remove copper from the body, include Cuprimine (penicillamine).…

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New Technology Found To Be Better Treatment for Hereditary Tyrosinemia Type 1 and Wilson Disease

LogicBio Therapeutics has just presented preclinical data of their GeneRide platform. This data demonstrates that the GeneRide technology is valuable in methylmalonic acidemia (MMA), hereditary tyrosinemia type 1 (HT1), and…

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New Data Available for GSD1a, OTC Deficiency, and Wilson Disease Studies
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New Data Available for GSD1a, OTC Deficiency, and Wilson Disease Studies

  In a recent press release, biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx") shared positive safety and efficacy data from Phase 1/2 clinical trials on gene therapy solutions for ornithine transcarbamylase…

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Website to Connect Liver Disease Patients To Clinical Trials
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Website to Connect Liver Disease Patients To Clinical Trials

According to NBC Right Now, the liver disease advocacy organization called The American Liver Foundation is launching an online platform designed to pair liver disease patients to clinical trials. The website,…

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Editor’s Choice: Adult Faces of Rare and Kids with SCID

Happy Friday Patient Worthians! We have two intriguing stories from PW contributors this week, detailing their plight with rare disease. Additionally, we have a pretty cool outcome for a pretty cool kid…

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Editor’s Choice: An Amazing Mom with MS & a Young Woman with Wilson’s

Welcome to this week's Editor's Choice! This week we are highlighting this inspirational, beautiful mother who wants to spread her message. She's currently battling multiple sclerosis in a courageous and proactive way,…

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