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…
The development of gene therapy as a treatment for rare and life-threatening conditions is a quickly growing area of scientific exploration. As more drug developers have begun investing in this…
According to an article originally published by HuffPost, an ambitious 34-year-old doctor is peddling plasma transfusions at steep prices, claiming a number of dubious health benefits. Ambrosia, LLC In 2014…
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…
According to a story from PR Newswire, the genomics medical company Sangamo Therapeutics, Inc., recently announced that the first patient had been dosed in its Phase 1/2 clinical trial which…
According to a story from EurekAlert!, the 60th Annual Meeting of the American Society of Hematology featured a number of different presentations of the latest research from St. Jude Children's…
According to a story from Reuters, the drug developer BioMarin is looking to make a name for itself in the hemophilia community in a very unique way. The company has…
Garret Schuster was just six months old when he was diagnosed with hemophilia. He went to see a pediatrician to treat an ear infection but the doctor sent him for…
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…
Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…
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 BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an…
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…
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