According to an article from ReliaWire, a sizable portion of the global population is likely to have an immunity to the inert viruses scientists use to administer gene therapy in…
According to a release from Sangamo Therapeutics and published by PR Newswire, experimental severe hemophilia A drug SB-525 proved to be well-tolerated and generally safe in a phase 1/2 clinical…
According to a story from BioSpace, the genomic medicine company Sangamo Therapeutics, Inc. and Pfizer, Inc. recently announced the release of interim data from a Phase I/II clinical trial. This…
According to a story from BioSpace, a recent study revealed that hemophilia A patients were less likely to experience bleeds when they used new, extended half-life therapies on a preventive…
According to a story from Hemophilia News Today, the drug developer Spark Therapeutics, Inc., has recently released several updates in regards to a number of its experimental gene therapies that…
According to a story from Scoop Independent News, citizens of New Zealand that have the rare bleeding disorder hemophilia A will soon have access to the drug emicizumab (marketed as…
As we age, we become more at risk for certain conditions. For instance, osteoporosis, atrial fibrillation, certain cancers, and heart disease, are more common in older individuals. These conditions pose…
BioMarin has just released an update on their achondroplasia and hemophilia clinical trials. Exciting things are in the works! Achondroplasia Update Achondroplasia causes short stature. A phase 3 trial working to…
Sangamo Therapeutics has shared details of their preliminary data from a clinical trial of the investigational drug SB-525 in patients with Haemophilia A. For more detailed information, you can read…
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…
A drug called Hemlibra that is currently only approved for treating a subset of patients with haemophilia A has been found to be effective for wider use, reports Hemophilia News…
According to a story from biopharmadive.com, the pharmaceutical company Roche is reporting a total of five deaths of patients with hemophilia A that have taken the company's product Hemlibra. While…
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…
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.…
Big news for those with hemophilia! On Thursday, the FDA approved Hemlibra (manufactured by Genentech), a weekly self-injection for hemophilia A patients who have developed resistance to standard medicines for preventing…
According to a late-summer press release sent out by the companies, two pharma players are joining forces and making waves in gene therapy for hemophilia A. Sangamo and Pfizer announce…
The Indiana Hemophilia & Thrombosis Center in Indianapolis has long been a leader in innovative patient care. They teamed up with MicroHealth, a company founded by hemophilia patient Aaron Craig…
It’s always exciting when two giants of their fields join together to accomplish something. When Pixar and Disney joined forces to bring us the best animated films since… ever, the…
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…
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