According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
According to a story from MedCity News, a patient with the rare blood disorder beta thalassemia will be the first patient dosed in a Phase I/II clinical trial involving a…
A recent publication from the Massachusetts Institute of Technology announced promising results from research into a novel new type of therapy called RNA interference (RNAi). RNAi could potentially be used…
According to a story from Market Screener, the biopharmaceutical company Marinus Pharmaceuticals, Inc. recently announced its plans to initiate a Phase 3 study that is intended to test ganaxolone as…
According to a story from dubainewstoday.blogspot.com, the data from a Phase I clinical trial suggests that an experimental drug candidate from the pharmaceutical company Boehringer Ingelheim could substantially improve the…
Happy Wednesday Everyone! We're excited for the spring weather coming up! Today, we're highlighting an article on some good news for the rare community in canada, followed by an update…
Medical devices include ECG electrodes, bandages, cardiac pacemakers, thermometers, and hemodialysis machines. A comparison of clinical trials shows that medical device trials are somewhat similar to pharmaceutical trials that evaluate drugs,…
About Pulmonary Hypertension Pulmonary Hypertension (PH) is a form of high blood pressure which affects the arteries. Primary Pulmonary Hypertension refers to a type of the condition whose origin is unknown. Secondary…
Hypertriglyceridemia Hypertriglyceridemia is the term used to describe abnormally high numbers of triglycerides in the blood. People with Hypertriglyceridemia have an increased risk of heart disease, stroke, and heart attack.…
According to a story from globenewswire.com, ProQR Therapeutics N.V. recently announced that it has begun dosing patients in its Phase 1/2 clinical trial. This trial is testing the company's investigational…
According to a story from BioSpace, the drug developer Horizon Pharma plc recently announced the release of topline data from a Phase 3 clinical trial of its experimental drug teprotumumab,…
As much as we try to minimize human error in the healthcare field, it still happens. To decrease the chance of its occurrence we’ve created various policies, rules, and guidelines…
According to a story from news-medical.net, a results of a recent study could help accelerate the development of therapies for liver diseases based on RNA interference (RNAi). This study demonstrated…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is the collective term used to describe ulcerative colitis and Crohn's disease. These conditions cause diarrhea, rectal bleeding, fatigue, weight loss, and rectal bleeding. These…
According to a story from the Breast Cancer Society of Canada, Veronica Dubois, who is currently pursuing a Master of Science in Medical Biophysics, is focusing her research on developing…
This week, a publication from BioSpace announced that a second individual has been cleared of HIV in another astounding success for stem cell therapy. The individual known only as the…
Wisconsin; the land of beer, cheese, good-hearted people, and Maria Voermans - rare cancer survivor and now fierce advocate for the rare disease community. Ten years ago, Maria had the…
According to a story from The Indian Express, doctors and drug developers are at a crossroads over an innovative form of therapy called fecal microbiota transplants. These transplants are essentially…
According to a story from Xtalks, there has been an uptick in research related to rare diseases and the development of new therapies for them. Despite this, only a small…
According to a story from news-medical.net, a team of scientists associated with the CURECN project are starting to develop a gene therapy that has the potential to cure the rare…
On February 24, 2019, the Organization for Rare Diseases India, USA (ORDIUSA) held a 7km walk/run at four different locations across the United States. The Event was called Racefor7 and its goal…
According to a story from Ottawa Citizen, the CHEO Research Institute has recently begun operations at its Centre for Genomic Innovation. Much of the the center's operations will focus on…
Happy Thursday! Today, we're highlighting an article on a story about rare disease discrimination, followed by news about a musician who went public with a rare diagnosis. After that, we…
"...just like 50,000 other people in the United States each year, this week I was diagnosed with stage 4 pancreatic cancer." Alex Trebek - who has has hosted the…
According to a story from Central Charts, the biopharmaceutical company Acceleron Pharma Inc. recently announced that the company's experimental drug candidate ACE-083 has earned Orphan Drug designation from the US…
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