According to a publication from CURE Magazine, in a phase 2 clinical trial nearly 50% of patients with a certain type of cholangiocarcinoma responded to a treatment comprised of two…
According to a publication Biospace, the American Food and Drug Administration has drafted a series of five new documents meant to better structure and organize future participation in cancer clinical…
According to a story from BioSpace, the biotechnology company Minoryx Therapeutics recently announced that the company has received official approval from the Spanish Agency of Medicines and Medical Devices (AEMPS)…
According to a publication from Multiple Sclerosis News Today, German biotechnology company Immunic Therapeutics has successfully enrolled the first participant for its phase 2 clinical trial for experimental relapsing-remitting multiple…
According to a publication from Impact Lab, clinical testing for a new kind of Alzheimer's treatment is slated to begin at Weill Cornell Medicine (Cornell University's medical school) in Manhattan…
According to a story from Markets Insider, the biopharmaceutical company Orchard Therapeutics recently announced the presentation of proof-of-concept data in regards to their investigational drug candidate OTL-102. This experimental therapy…
A study published in the Orphanet Journal of Rare Diseases monitored the long-term progression of 16 patients that had Niemann-Pick Disease type B. The study served to gather important observational data…
According to a story from Nursing Times, Claire Naisbitt is one of three multiple sclerosis specialist nurses that treat patients that reside in the Teesside community. She first took the…
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A recent announcement by the Ehlers-Danlos Society marks the beginning of the recruitment phase for an international study devoted to determining the root cause of hEDS. The gathering of this…
According to a story from BioPortfolio, Imbrium Therapeutics and Mundipharma EDO GmbH recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to their experimental…
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 recent article in the Globe, the Danish biotech company Saniona AB has initiated a Phase 2a clinical trial of Tesomet for the treatment of hypothalamic obesity, a rare…
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…
A recent article published in online CANCER NETWORK reports findings that indicate children who have survived Hodgkin lymphoma (HL) may develop solid tumors in later years. These findings will have…
According to a story from globenewswire.com, the drug developer The Cell Factory and the University of Padova in Italy have been conducting research in order to demonstrate the effectiveness of…
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…
Although some ultrasound and/or CT scan studies may show that patients’ lymph nodes (glands) are free of disease, upon further examination using sentinel lymph node biopsies,(SLNB) the doctors at MD…
What is Bronchopulmonary Dysplasia? Bronchopulmonary Dysplasia (BPD) is a diagnosis most commonly given to premature newborns. In children, BPD is also the most common cause of Pulmonary Hypertension (PH). Premature babies often develop…
Celgene Corporation has recently announced two exciting updates for the rare disease community! Update #1 The first big announcement from Celgene was that their supplemental New Drug Application (sNDA) for…
In November of 2018, England announced that cannabis could be prescribed for medicinal use by specialist physicians when no other treatments proved effective. It's prescription was purely to be decided…
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…
Tay-Sachs disease is a rare, genetic and sadly fatal condition. It's a neurodegenerative disorder caused by an impaired production of the β-Hexosaminidase A enzyme (HEXA). Symptoms of this condition include listlessness, diminishing…
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,…
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