Epidiolex was the first cannabis-based drug approved in the United States. It was first recommended for FDA approval by an advisory committee in April of 2018. In June of this…
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…
According to a story from Business Wire, the drug developer Bioverativ, Inc., recently announced its intention to present data related to its latest developments in blood disorder therapies. This data…
According to a story from PRNewswire, Infinity Pharmaceuticals and the Bristol-Myers Squibb Company recently announced that the two companies will be partnering in order to conduct a Phase 2 clinical…
The FDA has approved a new narcolepsy and cataplexy treatment for pediatric patients! Xyrem is a drug used to control narcolepsy symptoms including cataplexy and excessive daytime sleepiness (EDS). It…
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 Market Screener, the international pharmaceutical company Recordati recently announced that the European Commission (EC) has recently presented the company's experimental product candidate REC 0545 with…
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,…
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…
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…
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…
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 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.…
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…
Ellen Pompeo, who plays a doctor on Gray's Anatomy and was featured as a guest on Ellen Degeneres' The Ellen Show. We recently read an opinion piece on drjengunter.wordpress.com from OB/GYN,…
Any research that's done with rare disease patients in mind, working to improve their treatment and experience is wonderful. However, when collaboration between both researchers and organizations working to support…
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…
CNS-PNET brain cancers are typically diagnosed by type according to their specific location in the brain as well as how they appear underneath a microscope. However, its recently become clear…
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…
An innovative new treatment means practically nothing if we can't determine the type of patient it's going to be effective for. For an illness like acute myeloid leukemia (AML), which is…
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).…
According to a story from epidermolysisbullosanews.com, the biotechnology company TWi Biotechnology, Inc., recently announced that is has begun enrollment for its Phase 2 trial. This trial will test the company's…
In a recent statement, Scott Gottlieb, the Commissioner of the US Food and Drug Administration, has re-affirmed the organisation’s commitment to modernising drug regulatory pathways. New changes are hoped to…
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