Gene Therapy Could Change Hemophilia Treatment Forever
According to a story from CNBC, 53-year-old James Addie has been living with hemophilia since the day he was born. Because hemophilia is a genetic disease, the blood disorder is…
According to a story from CNBC, 53-year-old James Addie has been living with hemophilia since the day he was born. Because hemophilia is a genetic disease, the blood disorder is…
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…
According to a story from Business Wire, the drug developer Bioverativ, Inc., recently announced its intention to present data related to its latest developments in blood disorder therapies. This data…
According to a story from The Atlantic, the continual development of gene editing technologies like CRISPR have the potential to cure a substantial portion of rare diseases that are linked…
According to a story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…
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…
According to Science Blog, a therapeutics company is on its way to creating an implantable, insulin-producing device that may give injections a run for their money. Therapies similar to…
According to information provided by the National Hemophilia Foundation, people living with hemophilia now have the opportunity for a greater level of financial support. This new form of aid comes…
Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease. With fetal gene…
According to an article published on BioPortfolio, a charitable foundation known as The Assistance Fund recently began a program to assist with the medical costs of hemophilia. The Assistance Fund…
According to a story from the U.S. Food and Drug Administration (FDA), Commissioner Scott Gottlieb has released a press release which discusses in detail the agency's efforts in the development…
According to a June 20th report by the National Hemophila Foundation (NHF), the NHF along with several other prominent organizations in the bleeding disease community will be joining forces to…
Organisations are planning to develop new clinical practice guidelines for the diagnosis and management of von Willebrand disease. The original article can be read here, at PR Newswire (whose source…
According to a story from the National Hemophilia Foundation, a recent study examined the prevalence and impact of cardiovascular disease among hemophilia patients. It is widely assumed that people with…
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…
According to PRNewswire, Express Scripts announced a new program to benefit people living with rare diseases on May 31st. The program, called Second Opinion is part of the company’s larger…
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…
In order to keep up with the quick-growing field of gene therapies, the United States Food and Drug Administration (FDA) announced it will provide faster methods of approval for new…
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.…