According to a story from biopharmadive.com, the field of gene therapy has gained increasing relevance throughout 2019, and 2020 will likely be a continuation of this trend. An area of…
In a story for Hemophilia News Today, author Shellye Horowitz has written a piece titled "An Open Letter to Providers Treating Women with Hemophilia." Women still face discrimination and unequal…
According to a story from BioPortfolio, the drug company Octapharma USA sponsored the 71st Bleeding Disorder Conference, which is put on by the National Hemophilia Foundation. The company is also…
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…
According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients…
Camp Notaclotamongus In Eureka, Missouri, Camp Notaclotamongus has some typical activities for a summer camp. They state that camper goals aim to build friendships, confidence, and personal growth with outdoor…
Blood Donations For many people living with a rare disease such as Beta thalassemia, Hemophilia, Aplastic Anemia, Myelodysplastic syndromes, Acute Promyelocytic leukemia, and others, blood transfusions are not uncommon. For…
According to a publication from Reuters, based on early clinical data, American biotechnology company BioMarin Pharmaceuticals believes that a single injection of its experimental hemophilia A drug could relieve symptoms…
According to a story from CNBC, the now former Commissioner of the US Food and Drug Administration (FDA) Scott Gottlieb was recently featured on the news network for a discussion…
An article at ASH Clinical News recently detailed some of the history of genetic testing for bleeding disorders like hemophilia or von Willebrand Disease. Although true genetic screening has only…
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…
The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…
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…
"The simple idea of a single gene leading to a single disease is more likely to be an exception than a rule." Finding the Disease-Causing Genes Things would be, not…
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…
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A recent article in the National Hemophilia Foundation news quotes the Blood Transfusion journal's report on the results of a study conducted under the lead of Dr. Flora Peyvandi, Medical…
Rare diseases such as Tourette's syndrome and cystic fibrosis can be, among all of the other challenges that come with their diagnosis, extremely isolating. A recent article on Psychology Today explains…
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…
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…
People with spinal muscular atrophy and other rare conditions with either few or no approved treatments have recently had their hope of a cure renewed by the concept of gene therapy. Basically,…
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…
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