According to a story from EurekAlert!, the results from a recent Phase 2 clinical trial suggest that a two drug combination could be a useful approach for people with rare,…
According to a story from BioPortfolio, the gene therapy company Rocket Pharmaceuticals has recently announced that the company's investigational gene therapy RP-L102 has earned both Fast Track and Regenerative Medicine…
According to a story from Reuters, the drug developer BioMarin is looking to make a name for itself in the hemophilia community in a very unique way. The company has…
According to a story from statnews.com, the recent FDA approval of a new treatment for Lambert-Eaton myasthenic syndrome could actually be bad news for patients. As it turns out, the…
According to a story from Grimsby Live, the family of six year old Melissa Stark, who has Williams syndrome, has transformed their house into a Santa Claus themed wonderland of…
According to a story from Medical Xpress, a team of scientists affiliated with the University of Granada have developed a new potential treatment for a form of mitochondrial disease that…
According to a story from Science Alert, an ugly and unpleasant tale involving the devastating rare genetic illness known as Huntington's disease will culminate in a lawsuit. The outcome of…
According to a story from Xconomy, the drug developer Vertex Pharmaceuticals recently announced that a three drug combination treatment for the rare lung disorder cystic fibrosis has performed well in…
According to a story from PR Newswire, the biopharmaceutical company Constant Pharmaceuticals recently announced that it plans to develop its experimental compound TXA127 as a therapy for epidermolysis bullosa (EB).…
At Patient Worthy, we are dedicated to stories about the latest in rare disease news and research, as well as posting stories from rare disease patients themselves. However, there are thousands…
The US Food and Drug Administration recently announced the approval of the first biosimilar drug for patients with non-Hodgkin lymphoma in a press release. The drug is known as Truxima…
According to a story from accesswire.com, the drug developer BioVie, Inc., recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to the company's investigational…
At Patient Worthy, we are dedicated to stories about the latest in rare disease news and research, as well as posting stories from rare disease patients themselves. However, there are…
According to a story from Digital Journal, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that its investigational drug candidate ACE-083 was granted Fast Track designation from the US Food…
According to a story from BioPortfolio, Celgene Corporation and bluebird bio, Inc., have recently announced that enrollment for a pivotal Phase 2 study testing an experimental therapy for relapsed/refractory multiple…
According to a story from Buzzfeed News, Stephen Hillenburg, the creator of the smash hit children's cartoon SpongeBob SquarePants has passed away at age 57. Last year, Hillenburg had revealed…
According to a story from MarketWatch, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., recently presented interim data from a Phase 2 trial of setmelanotide as a treatment for obesity associated with…
According to a story from Parkinson's News Today, more and more research is indicating the exercise can have many significant benefits for Parkinson's disease patients. While there are no therapies…
According to a story from globenewswire.com, the biopharmaceutical company PhaseBio Pharmaceuticals has recently announced that the first patient has been dosed in a Phase 2b clinical trial of the company's…
According to a story from The Star Online, the government of Malaysia has allotted RM50 million of its 2019 budget for the treatment of numerous rare diseases, stunted growth, hepatitis…
According to a story from finanznachrichten.de, the UK drug development company AstraZeneca recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation for the company's medical…
According to a story from pm360online.com, the drug developer Novartis has recently announced that the company's gene therapy Luxturna has gained approval in the EU as a treatment for Leber's…
The following story includes discussion of suicide. According to a story from EurekAlert!, a recent study has revealed that survivors of head and neck cancer are four times more likely…
According to a story from The British Journal of Cardiology, the use of renin-angiotensin-aldosterone-system (RAAS) inhibitors or blockers is a valuable component of treatment for many medical conditions and diseases,…
According to a story from Business Wire, a recent survey has revealed new findings about the frequency of neurogenic orthostatic hypotension (nOH) among Parkinson's disease patients. Around one in five…
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