The first patient has been dosed in the Phase 1/2 B-LIEVE trial of FLT180a, an investigational treatment for hemophilia B. Freeline Therapeutics, the drug developer, announced the news in a…
In a recent article for Hemophilia News Today, Jennifer Lynne, who is diagnosed with both von Willebrand disease and hemophilia B, stresses the importance of remembering women can have hemophilia…
According to an article from the National Hemophilia Foundation, CSL Behring and uniQure have recently provided an update on their phase III HOPE-B trial. This study is evaluating etranacogene dezaparvovec,…
According to Hemophilia News Today, GENV-HEM, an investigational treatment for patients with hemophilia A and B either with or without clotting factor inhibitors, earned Orphan Drug designation from the FDA.…
According to a story from the National Hemophilia Foundation, the drug company uniQure has provided an update in regards to a case of hepatocellular carcinoma (HCC) that emerged in a…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
By Lauren Thayer from In The Cloud Copy Hemophilia is a medical condition in which a person’s blood clotting ability is severely reduced. This inability to properly clot leads to…
According to Hemophilia News Today, data collected from a Phase 2b trial highlights that the subcutaneously administered DalcA is effective for the treatment of hemophilia B. Find the full press…
The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…
According to a story from Hemophilia News Today, the drug developer uniQure has completed its goals for patient enrollment ahead of time for is phase 3 clinical trial. This clinical…
According to a story from MD Edge, a phase 2 clinical trial testing the drug concizumab as a treatment for hemophilia A and hemophilia B demonstrated substantial capability in reducing…
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.…
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