Full Patient Enrollment in Study Expected by Year-End 2019 LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2019 -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for…
According to a story from hemophilia.org, the drug developer uniQure recently announced that the first patient had been dosed in the company's Phase IIb clinical trial. This trial is testing…
Hemophilia can affect anyone. Let’s make a few things clear: Women are carriers of hemophilia and pass the gene, but men determine the child’s gender. It’s not all on you,…
To the patient: Accepting your diagnosis doesn’t mean you can’t be mad about it. It simply means you understand that the reality of your life has changed and you know…
Dear Parents, You need to understand that you are taking on a completely new role in your child’s life. You will no longer be controlling their hemophilia for them. It’s…
Why is non-adherence still a problem for many hemophilia patients? There’s a very simple answer here: it’s because hemophilia can be difficult to manage and hard to live with. There’s…
We often hear the term noncompliance when talking about patient-doctor relationships for individuals with hemophilia. In essence, it means the hemophilia patient hasn’t done what the doctor has asked them…
In the Victorian period, the British Royal family was known to carry haemophilia, leading to the condition also being known as ‘the royal disease’. Now, no members of the British…
According to a story from eurekalert.org, a recent test study suggests that patients with hemophilia B could be treated with just a single injection over the course of their lives.…
United in Progress is the tag line for a unique collaborative effort to speed progress in identifying, understanding and treating hemophilia: My Life Our Future. Increasingly in the area of rare…
According to a story from the National Hemophilia Foundation, the pharmaceutical company Novo Nordisk announced that its product REBINYN was now available for hemophilia B patients. The treatment is intended…
Many people aren’t aware that even if they have their emergency medication with them, with instructions, that it may not be given to them. Their carefully labeled, lifesaving treatment could…
A huge deal is underway as Sanofi looks to acquire Bioverativ for $11.6 billion with hope to enhance and strength their rare disease presence, reports Genetic Engineering & Biotechnology News.…
Pharmaceutical company and gene therapy leader, uniQure, published promising data from their current phase I/II trial testing out a gene therapy called AMT-060 in patients with serious cases of hemophilia B.…
A perfectly coifed woman-child cuts you off on the way to the YMCA. Your children are copping some major attitude. Your spouse forgets to grab milk for dinner. Do you…
Patient Worthy attended the National Hemophilia Foundation's 69th Annual meeting in Chicago and we are thrilled to report on some of the exciting new developments for patients with bleeding disorders.…
Gene therapy is rapidly shaping up to be the next stage in the evolution of treatment for a wide array of conditions. And it makes sense: rather than treat the…
Hema Biologics have recently released something even cooler and more inventive than just their traditional hemophilia treatments. They are way on top of their game and know exactly how to…
We take most of our daily activities for granted. From working out at the gym to having our teeth cleaned, but for people living with hemophilia B what most people…
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Let's talk about Mount Everest. It sits on the border between Nepal and China and its peak is 29,029 feet above sea level--which makes it the highest mountain on earth.…
There are few things more frightening and horrifying for a new parent than being told that their child has a lifelong medical disorder. Perhaps the only words that could make…
Happy Friday Patient Worthians! April is Sjogren's Awareness Month! There was also a whole day dedicated to Bohring-Opitz Syndrome. The MDS Foundation has another upcoming event as well. Lastly, last month…
Royalty, murder, lots and lots of blood – sounds like elements from the plot of a Victorian-era thriller. Actually, this story comes straight from hemophilia history. Science Magazine published a…
Welcome back to Editor's Choice! This week we have an interesting take on Parkinson's treatment. We also have an empowering article from one of PW's rare disease contributors. We are all feeling the…
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