Happy Friday! Winter is coming and so is FDA news! Take a moment to check out some recent FDA announcements. FDA Approves Triplet Immunotherapy Combination for Multiple Myeloma Big news…
Are you interested in learning about the latest trends and drug development in the rare disease field? Then you should check out a special Journalist Team Presentation from Global Data…
According to a story from telegram.com, a group of researchers from the University of Massachusetts medical school recently announced that they may have reached a major treatment breakthrough for genetically…
Society teaches us to make comparisons all of the time. When you chose to participate in a competitive game, and you want there to be a winner and a loser,…
While I write about rare and neglected disorders all the time, very few people in my life actually know about my own experience. I have hypermobile Ehlers-Danlos syndrome (EDS), a…
In a recently shared article, the University of Southern California has announced that the United States Food and Drug Administration has approved a high-tech brain scanner at the Keck School…
According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has given the company's experimental product PD-0325901 Orphan Drug…
My name is Jasmine Taylor and I am 12 years old. October is one of my favorite months (July is the other one- it’s my birthday month!)… Let me tell…
A new study into the potential use of a tau-PET tracer for Alzheimer’s disease has produced promising results. The data was presented at the Clinical Trials on Alzheimer’s Disease Conference,…
A recently published report on neuromuscular disease care outlines the current challenges and future opportunities in diagnosis and treatment. The report, titled ‘Understanding Neuromuscular Disease Care’, was funded by the…
According to a story from Scleroderma News, many rare disease patients will be heading to the polls on November 6th. Many of them are as deeply concerned about the high…
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.…
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…
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…
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…
Goethe University in Frankfurt, Germany has been awarded a grant of more than one million euros as part of a large-scale project in Europe to standardise and make available clinical…
The Cambridge Rare Disease Network’s RAREfest, a festival focusing on rare diseases, will bring together researchers, scientists, patients, and the public. It’s taking place on the 30th November and 1st December in…
Novartis has announced positive findings from their research into brolucizumab, an investigational drug being developed to treat neovascular age-related macular degeneration. This data comes from year two of the…
This is the second part of Amber's story. Check out part one here. June rolled around, and my appointment with the rheumatologist came up. The hour-long car ride there made…
For esophageal cancer (like many rare conditions), early diagnosis means a better prognosis. Unfortunately, until now there haven't been many ways to determine whether or not an individual may be at…
November is Pancreatic Cancer Awareness Month. The American Cancer Society’s estimates for pancreatic cancer in the United States for 2018 are: About 55,440 people (29,200 men and 26,240 women) will…
Hello all! My name is Amber Aerni. I am 28 years old, and I would like to tell you the story of my POTS journey. Yes, I refer to my…
According to a story from globenewswire.com, the drug developers Pfizer and Novartis have recently announced the beginning of a collaborative effort to test new potential therapies for nonalcoholic steatohepatitis (NASH).…
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