According to a story from Angioedema News, the results of a recent study have led authors to conclude that genetic testing can be useful for the diagnosis of hereditary angioedema…
Of all possible symptoms, four year old Alex Vasey developed one that most people don't associate with disease. As originally reported in The Daily Record, Alex’s rare genetic condition, Williams…
Sometimes through tragedy and hard times, a beacon of hope can emerge. After months of struggling with a rare aggressive disease, toddler Chloe Barnes passed away in 2008 in Minnesota.…
According to a story from BioSpace, the genetic medicines company Passage Bio recently announced plans for its sixth program for gene therapy development. The company plans to work with the…
We're almost halfway through September believe it or not! As autumn approaches, we're continuing the aggregate rare disease news. This week, we're highlighting three studies, and one article on a…
One of our partner organizations, the MDS Foundation, is hosting another free MDS Patient & Family Forum in conjunction with Kansas University Medical Center, with support from Celgene, Novartis, Takeda,…
According to a story from journals.lww.com, The Lazarex Cancer Foundation and the University of California San Francisco have announced plans to work together on a three year study that will…
According to a story from Biotech 365, the biotechnology company Viela Bio announced the publication of critical results from a recent study that tested the company's investigational drug inebilizumab as…
According to a story from meaction.net, Jaime Seltzer, the Director of Scientific and Medical Outreach at #MEAction, recently spoke at the Third Annual Working Group Meeting on the Molecular Basis…
According to a story from firstworldpharma.com, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the company's experimental product candidate sotatercept has earned Orphan Drug designation from the US Food…
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According to a recent article in MedicalXpress, the newest discovery in the search to reduce the risk of AD was through the work of researchers led by Dr. Jaehong Suh,…
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The fountain of youth may actually be in a cocktail of drugs that were part of a new study on aging. A recent article in Longevity’s Science Research announced…
According to a story from The Rheumatologist, evidence suggests that patients with rare diseases, such as autoimmune diseases, can benefit from visiting combined clinics, which are special medical centers in…
In the US, medical costs come in the form of drug prescriptions, doctors visits, expensive surgeries, and visits to specialists. As originally reported in The Tyee, in Canada, patients are…
According to a story from Forbes, a new drug developed by the company Regeneron has the potential to make a real difference for people with the homozygous form of familial…
According to a story from rheumatology.medicinematters.com, a recent Italian real-world study has provided some evidence that the drug apremilast, which is considered a phosphodiesterase-4 inhibitor, could be a useful treatment…
According to a story from the Washington Post, the University of Virginia (UVA) Health System is often ranked as the best in the Old Dominion, but it also pursues debts…
According to a story from Columbia University, the country's All of Us Research Program is making consistent process in its ultimate goal to enroll a minimum of 1 million participants. These…
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According to a recent article in The Scientist, Northwestern Medicine’s successful autoimmune and immunotherapy program is winding down and will be closed by the end of the year. The program,…
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…
Rare diseases often lack a voice. Unlike more common diseases, they do not have big movements that bring the disease awareness through fundraisers, 5Ks, and spokespeople. However, sometimes, celebrities have…
According to a story from BioPortfolio, Alexion Pharmaceuticals, Inc. and Eidos Therapeutics, Inc., have recently struck an agreement related to the development and marketing of an experimental treatment for hATTR…
According to a story from Medical Xpress, a recent study has revealed that a certain genetic premutation affected the FMR1 gene could be a risk factor for far more health…
According to a story from EurekAlert!, a team of scientists affiliated with the Harvard Medical School and the Joslin Diabetes Center recently published a study that is giving researchers the…
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Pharmacogenomics plays an important role in predicting whether patients will benefit from certain medications or if the medication will cause serious side effects. The process involves the way in…
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