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 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…
According to a story from the Hindustan Times, seventeen year old Yash Galpelli was born with Wilson's disease, a rare genetic disorder. For a long time, he had no idea…
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…
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,…
Medical malpractice. These two words strike fear into the hearts of millions of doctors all over the world, but especially in litigious societies, such as the United States. Not all…
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.…
Wilson disease is a rare genetic disorder. What does it cause? Well, copper begins to build up in the liver and brain, sometimes to a dangerous degree. One young boy in India…
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…
Alicia Goss is a college student, and for a long time she didn't know she had a rare condition called Wilson disease. She just wanted to go out and have…
Wilson’s disease is a genetic disease that may cause copper build up which effects both the brain and liver. Wilson’s disease affects about 1 in 30,000 people in the world.…
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 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…
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,…
Emma Gaul’s story is a good reminder of how quickly life’s plans can change when your body turns against itself. Less than two years ago she was a vibrant and…
Would you climb 1,776 steps to raise money for charity? What if you were in a wheelchair? Julian Backhouse is in a wheelchair, but that isn’t stopping him from climbing…
Happy Friday Patient Worthians! This week, we have some riveting stories by three PW contributors. Firstly, we have Alexis battling Lyme disease for 8 years and a surprising ending to Part 1…
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…
Wilson Disease: the rare, autosomal (meaning it occurs in both men and women), recessive (meaning that an individual must inherit one copy of the mutated allele from each parent to…
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