The United States Food and Drug Administration has awarded Orphan Drug Designation to an experimental PLX cell therapy (called PLX-R18) as a treatment for graft failure and incomplete haematopoietic recovery…
According to a story from Greenville Online, the Greenville Health System has begun implementing a comprehensive treatment program for patients with sickle cell disease and its most severe and common…
According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug…
Happy Friday, everyone! We're trying something new this week. Instead of wrapping up with an Editor's Choice article, we're highlighting four FDA announcements we think you should know. Check them…
The United States Food and Drug Administration has awarded twelve research grants worth a total of over $18 million for research into rare diseases. For more information about this news,…
According to a story from BioSpace, the biotechnology company BioElectron recently announced that the results from its Phase 2a clinical trial of its experimental product EPI-589 are looking positive. EPI-589…
According to a story from UPI, gene editing technology could be a potential treatment for mitochondrial diseases, a rare, genetic illness that is usually fatal. In a proof-of-concept experiment, researchers…
Show of hands - who has ever indulged in a little beauty pampering? A mani/pedi? A haircut? A shave? Sure, that was a trick question - who hasn’t?! And that’s…
Just Searching for Answers When diagnosed with a rare disease, cancer, or other illness, it makes sense that you’d research every type of treatment on the market. However, there are…
According to a story from Angioedema News, an investigational product from BioCryst Pharmaceuticals called BCX7353 was able to quickly subdue swelling attacks in hereditary angioedema in clinical trials. In addition,…
October 1st is International Gaucher Day! What is Gaucher Disease? Gaucher disease is a lysosomal storage disorder in which the activity of the enzyme beta-glucocerebrosidase is incredibly low or non-existent.…
According to a story from globenewswire.com, the pharmaceutical company Portola Pharmaceuticals recently announced that its investigational product cerdulatinib has earned Orphan Drug designation from the US Food and Drug Administration…
According to a story from lupus.org, a recent spending package was passed in the US House of Representatives that includes $14 million in funding specifically for researching lupus, an autoimmune…
Attitude as defined in the dictionary: “A settled way of thinking or feeling about someone or something, typically one that is reflected in a person’s behavior.” In my opinion, how…
Sangamo Therapeutics has shared details of their preliminary data from a clinical trial of the investigational drug SB-525 in patients with Haemophilia A. For more detailed information, you can read…
According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold.…
A study carried out by a research team at Yale University and mainly funded by the National Institutes of Health has identified a cell in the spinal fluid of patients…
According to a report by medicalexpress.com, researchers recently discovered a new genetic condition. Though researchers previously described the condition in animals, no human evidence had yet been found. Michigan State…
After their own personal struggles with cancer, the Rendina family are working to raise money and awareness to fight the disease. For more information, you can view the source press…
Researchers at the University of Pennsylvania have studied the genetics of a class of neurological disorders that includes Huntington’s disease and Fragile X Syndrome. They found that there were similarities…
Michael Dickerson, aged 42, is originally from Hoedspruit Limpopo, South Africa. Recently, Michael was participating in a series of long distance trail runs when he learned about the story of Daniël…
Maurice Elias, who was born with severe combined immunodeficiency, has met up with the doctor who saved his life almost fifty years ago. For more information, you can read the…
According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial…
According to a story from Business Wire, the biotechnology company Abivax recently released the results from its Phase 2a clinical trial. This trial was testing its investigational product ABX464 as…
According to a story from healthnewsdigest.com, teams of researchers from around the world have successfully revealed a new biomarker that can help predict treatment outcomes in ovarian cancer. The activity…
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