In a recent press release, Knopp Biosciences announced that their drug therapy KB-3061 was granted Rare Pediatric Disease Designation to treat patients with KCNQ2 epileptic encephalopathy (KCNQ2-EE), a rare…
For twenty-five years Women of Influence has focused on empowering and helping to advance women in the workforce. Maayan Ziv, a Canadian tech entrepreneur, disability advocate, and voted one…
A duodenoscope is a device used in endoscopic retrograde cholangiopancreatography (ERCP) procedures, in which a doctor uses them to see the top of the small intestine. For a long time,…
Results of a Phase III study of CC-486 (oral form of azacitidine) showed a significant improvement for older patients who had been transfusion dependent. The results were favorable when…
Khevin Barnes is a cancer survivor, as he won his battle with breast cancer. While less than one percent of breast cancer diagnoses are males, it is very important to…
At the start of 2020, we had no idea what this year would bring: COVID-19, a novel coronavirus declared a pandemic in March. As of the morning of July 17,…
This past week, biopharmaceutical company Kiniksa Pharmaceuticals announced that their drug, rilonacept, received Orphan Drug Designation from the FDA. Following positive data from the RHAPSODY Phase 3 clinical trial,…
According to the American Cancer Society, there are approximately 1.7 million people in the U.S. diagnosed with cancer each year. About forty percent of these cases may be eligible…
According to a story from Fabry Disease News, any member of the Fabry disease patient community should strongly consider signing up for the Fabry Registry. Getting involved comes with several…
In the GRAPPA 2020 Annual Meeting, held virtually this year, Dr. Philip Helliwell acknowledged that he, like many rheumatologists, struggled to understand a certain condition: SAPHO syndrome. According to…
According to a story from Fabry Disease News, a recent case study analyzed the situation of two cousins living with Fabry disease who were being treated with enzyme replacement therapy,…
According to a story from BioSpace, the biotechnology company Ascentage Pharma has just announced that it has been given Orphan Drug designation from the US Food and Drug Administration (FDA).…
Happy Friday! We're more than halfway through July. Where is the summer going? As a reminder, it's Juvenile Idiopathic Arthritis Awareness Month and if you're looking for ways that you…
According to a story from Star Tribune, Chelsea Coenraads was 14 months old when her mother Monica became convinced that something seriously wrong with her daughter. She had failed to…
According to a story from theindychannel.com, brothers Elias, age eleven, and Emmett, age seven, were both born with the rare bleeding disorder hemophilia A. However, this has not stopped them…
Orphan drug designations are granted by the FDA to drugs and biologics that are developed for the treatment of rare diseases. According to a recent article in Biospace, Oncternal…
In the early 1990s, Dean Kamen was at the mall when he came up with an idea. After seeing a man in a wheelchair struggle to be noticed by…
According to a story from Korea Biomedical Review, Seoul National University Hospital (SNUH) in South Korea has just announced recently that the process of recruiting patients for the National Bio…
In 2014, Ki’tay D. Davidson passed away at the age of 22. A black, transgender activist, Ki'tay also used his voice to amplify the experiences and stories of people…
Living with a rare disease can come with some difficulties, whether those are physical, mental, or financial. It is estimated that 30 million Americans live with a rare disease, a…
According to a story from PR Newswire, new international treatment recommendation guidelines have recently been updated for nontuberculous mycobacterial (NTM) lung disease. The update pertains to a variant known as Mycobacterium…
According to an article in Globe NewsWire, Onconova Therapeutics, a biopharmaceutical company currently focusing on myelodysplastic syndromes, recently announced results of its Phase 1 study of oral rigosertib in…
According to a story from keloland.com, Westin Cuka, age six, was first diagnosed with neurofibromatosis, a rare disorder, three years ago. Since then, the family has traveled on multiple occasions…
Rare pediatric disease designation has just been granted to Rhythm Pharmaceuticals for their investigative therapy called setmelanotide. This drug is being investigated as a therapeutic option for two conditions- leptin…
In a July 9 press release, the Familial Chylomicronemia Syndrome (FCS) Foundation announced its support for a bill introduced by Congressman Paul Tonko (D-NY) and David B. McKinley (R-WV).…
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