What can you do with half an hour? That’s the question that a new Canadian program asks. An online program called #30MinutesThatMatter gives Canadian patients an opportunity to share how…
If drugs were measured by the quantity of approvals, orphan drugs would make quite a splash. Measured in more financial terms, however, they make up a much smaller portion of…
According to a story from BioSpace, the pharmaceutical company Advicenne has been granted clearance from Health Canada's Office of Clinical Trial to extend its Phase III clinical trial, which is…
According to a story from Science Daily, a group of scientists from the Australian National University have managed to discovery a new genetically linked disease and have also pioneered a…
According to a recent press release by Anavex, the United States Food and Drug Administration has approved an investigational new drug application for a study of the drug Anavex®2-73 in…
According to a story from BioSpace, the orphan liver disease drug company Albireo Pharma recently announced that its investigational product A4250 was granted Orphan Drug designation by the US Food…
According to a story from financialbuzz.com, the biopharmaceutical company Principia Biopharma recently announced that its experimental medical product PRN1008 has received Orphan Drug designation from the US Food and Drug…
According to a story from BioSpace, the biotechnology company Cellenkos, Inc., recently announced that the US Food and Drug Administration (FDA) has given the company the go ahead to begin…
According to a story from Ottawa Citizen, the Pan-Canadian Research Network on Lyme Disease recently received a boost of $4 million from the Canadian government and The Canadian Institutes of…
The 2018 FH Global Summit featured fantastic speakers, timely information, and valuable insight about the past, present and future of the way FH is understood, diagnosed, and managed. The summit…
According to a story from businesses.com.au, a new blood test that is currently in clinical trials could more accurately determine which patients will need chemotherapy to cure their cancer after…
According to a story from markets.businessinsider.com, the drug development company Pharming Group N.V. recently announced results from a Phase II trial testing its medication RUCONEST. This drug was tested as…
Genomic testing is becoming increasingly common, but more work still needs to be done into understanding which gene variants are linked to health and disease. To address this, the National Institutes…
According to a story from Healio, Dr. Monica Konerman recently gave a presentation about the importance of lifestyle changes in treating nonalcoholic fatty liver disease and its rarer and more…
The companies Genentech and Roche are working together to carry out a program of studies involving patients with Huntington’s disease (the huntingtin-lowering trial ASO program). One is a natural history…
This is a video presentation I created called Living Well with Dystonia. It shares some of the many things I have learned in my almost 20 years of living with…
The United States Food and Drug Administration has granted the drug Lynparza® (olaparib) Orphan Drug Designation for the treatment of pancreatic cancer. Lynparza is used in the US to treat…
You may know her from movies like Legally Blonde, Cruel Intentions, and Hellboy - but now Hollywood star Selma Blair has a new role she's tackling: multiple sclerosis advocate. Just…
The United States Food and Drug Administration has granted Fast Track designation to a drug being researched as a potential treatment for progressive familial intrahepatic cholestasis, a serious liver disease…
Across the globe, countries are transitioning their healthcare systems to provide more care within the patient's home and less in a hospital setting. Why? Simply put, it's because there's beginning…
British Columbia faces excruciating decisions about how and whether to cover drug treatment for rare diseases. One particular drug is Spinraza. It is a drug that is a part of…
The United States Food and Drug Administration has awarded Rare Pediatric Disease Designation to a potential drug for Mucopolysaccharidosis type I. For more detailed information about this news, you can…
Multiple sclerosis is a disease that can effect every aspect of daily life. However, symptoms vary on a day to day basis and the lack of predictability makes the disease…
Happy Friday everyone! The leaves are beginning to change, and so are ongoing activities at the FDA. This week, we're highlighting four FDA announcements we think you should hear about.…
This story contains images of severe lesions and blistering. On the weekend of October 12-14th of 2018, Patient Worthy had the privilege of attending the International Pemphigus & Pemphigoid Foundation's …
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