A study has found that a class of drugs known as MDM2 inhibitors may have the potential to help patients with ocular inflammation uveitis, a serious condition that affects the…
Alnylam has provided updates about their programs for patients with primary hyperoxaluria type 1 (PH1), a rare condition that affects the kidneys and bladder. As part of their Alnylam Act® program,…
Late-onset Pompe disease is associated with many features, one of which is a change to the voice, which may be noticeable in speech and voice quality. To investigate how this…
According to a story from Newswire, the specialty pharmaceutical company Tikomed recently announced that the first patient has been dosed in an clinical trial testing the company's experimental product ILB.…
The 2018 Nobel Prize for Medicine was awarded to two researchers working on checkpoint inhibitor therapies for cancer. A source article by AFP, shared by medical express, outlines how checkpoint…
According to a story from the Evening Times, Rebecca Scott was only in her upper twenties when she was first diagnosed with ovarian cancer. It is generally uncommon for ovarian…
According to a story from abc.net.au, Cyril Wise watched in horror as his wife Kaye died of Huntington's disease in 2014. However, before his wife's death, Cyril was struck with…
According to a story from pm360online.com, the drug developer Bavarian Nordic A/S recently announced that its Phase 2 clinical trial of its experimental product BN-Brachyury has begun, with the first…
Forbes reports that Healx, a British biotech startup, is using the power of artificial intelligence to find new uses for existing drugs that treat rare diseases. "Healx combines AI technology, pharmacology…
A study, called ‘Prevalence of Variant Reclassification Following Hereditary Cancer Genetic Testing’, has examined how often the results of genetic testing for cancer risk are later reclassified. To read about…
An enormous anonymous donation of more than $18 million has been given to the BC Cancer Foundation to fund cutting-edge treatments. The news was announced in an article by the…
It's shocking and quite frankly, devastating, to be diagnosed with any type of cancer at any age. It can be hard to stay positive as your life completely changes as…
According to a story from publicnow.com, the drug development company Sarepta Therapeutics recently announced that is has completed negotiations for a licensing agreement with Lysogene, a biopharmaceutical company. This agreement…
According to a story from The Sydney Morning Herald, the Australian government recently announced that it will be adding Galafold, a drug used to treat rare Fabry disease, to the…
A study funded by the Lustgarten Foundation, the largest private funder of pancreatic cancer research in America, has shown promising results. The scientists are hopeful that their work could lead…
According to a story from ScienceNews, a recent study has revealed an unusual connection between the appendix and Parkinson's disease. This research found that people who received an appendectomy (surgery…
Happy November! As we recover from Halloween and prepare for Thanksgiving, take a moment to check out some recent FDA announcements. FDA grants breakthrough therapy designation to UGN-101 for urothelial…
According to a story from BioPortfolio, the biopharmaceutical company GenSight Biologics recently announced that the first patient has received treatment in a Phase I/II clinical trial testing the company's experimental…
According to a story from The Daily Texan, professor Marian Williams-Brown from the University of Texas is conducting research in an effort to develop a more effective approach for detecting…
According to a story from Business Insider, two researchers from Stanford University, Lawrence Steinman and Jonathan Rothbard, have decided to start a new company called Katexco Pharmaceuticals. This startup will…
A recent article published by the University of California San Francisco looks back at the development of gene therapy technology and explores how scientists at the university are working to…
RAREfest, an event organised by the Cambridge Rare Disease Network (CRDN), will bring together patients, scientists, researchers, clinicians, support groups, businesses, and the public, to engage and educate people about…
According to a story from Biospace, the biopharmaceutical company Ultragenyx Pharmaceutical Inc. recently reported that its experimental product UX007 failed to achieve its primary endpoint in a recent clinical trial.…
According to a story from bioengineer.org, a new bacterial imaging tool has been deemed to be safe and practical device for used in the medical setting, a recent study claims.…
According to a story from Medical Xpress, a recent study suggested that the targeted cancer drug olaparib could be an effective treatment for women with ovarian cancer that has been…
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