According to a story from PR Newswire, presentations at the 60th annual American Society of Hematology Meeting and Exposition are highlighting the results of four large scale clinical trials that…
According to a story from BioSpace, the biotechnology company Tiziana Life Sciences Plc has recently announced that participant enrollment in its Phase 2a clinical trial has finished. This clinical trial…
According to a story from einnews.com, the cell therapeutics company American Gene Technologies (AGT) has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation…
In the UK, a think tank determines access to rare disease drugs based on their perception of an individual's quality of life. That idea has also been developed in the…
According to a story from BioPortfolio, the drug developer Novartis recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational gene therapy AVXS-101, also known…
According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company's latest…
According to a story from The San Diego Union-Tribune, the Pancreatic Cancer Prevention and Screening Clinic at UC San Diego Moores Cancer center has opened for the first time this…
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 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…
Tiffany Rowe was 15 when she accomplished her biggest childhood dream. She met Michael Jackson at a live concert and actually got to dance on stage with him. Over…
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 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).…
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…
For many families, when their child is born with developmental delays, there may be weeks, months, or even years that go by without a diagnosis. During this time, parents…
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…
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 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…
Just this week, the Food and Drug Administration approved a drug that targets a wide range of cancers based on DNA, rather than where the tumor is in the body. This is being…
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…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
