According to a story from The Print, Neha Shoriwala, a mother from New Delhi, is urgently seeking help for her son Harshil, who lives with Wilson's disease, a rare illness…
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).…
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…
Clinical trials can be beneficial for finding and evaluating new treatment options. Recently, biopharmaceutical company AstraZeneca shared how ALXN1840, a potential treatment for patients with Wilson disease, reached its primary…
According to a recent press release from Pfizer and biotechnology company Vivet Therapeutics, VTX-801, a gene therapy candidate for patients with Wilson disease, recently received Fast Track designation from the…
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…
According to a story from Globe Newswire, the biopharmaceutical company Ultragenyx Pharmaceutical Inc. has recently announced that its investigational treatment UX701 has earned the Orphan Drug designation from the US…
According to an article from BioSpace, Vivet Therapeutics and Pfizer have entered into a manufacturing agreement to develop Vivet's gene therapy, which treats Wilson disease. The terms of the agreement…
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A recent article in Forbes highlights a study on diagnostic performance published in The Lancet Digital Health. The study represents the first review to compare the diagnostic accuracy of deep…
Famous poetic thought suggests that "the eyes are the window to the soul." Doctors studying Wilson disease, however, may see the eyes as the window to a diagnosis. According to…
Despite a politician’s recent explanation of supply and demand by saying, “If you put the supply out there, the demand will follow,” that is not how the economic principle works.…
The liver is a pretty important organ. Some people think of it as the place where alcohol goes. Your neighbor’s kid in the 6th grade will tell you that it…
Wilson disease is one of those conditions that creeps up on the patient. The disease causes the body to be unable to properly metabolize copper, which as a result, builds…
We hear of feuding nations: Countries that share a border, yet can't get along with each other. So, it's heartening when I read of a young girl in Pakistan who…
Happy Fall Friday Patient Worthians! This week we have the amazing story about a woman with dystonia. We also have a video review of a man diagnosed with CVID. Additionally,…
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…
The WDA have over 1150 participants for this year's Big WOW! happening October 1st in cities all across North America. If you haven't had a chance to sign up, now's the…
Rare diseases often go undiagnosed because they are, after all, rare. A rare disease isn't usually the first thing anyone thinks of, in light of certain symptoms. It's only when doctors know what…
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