According to a story from Fabry Disease News, the drug developer Avrobio recently announced that the US Food and Drug Administration (FDA) has given the company's experimental drug AVR-RD-01 Orphan…
What is the Disparity? America is greatly divided by many things and race has always been one of them. Not only are there large social and economic divides among racial…
Canavan disease is a type of leukodystrophy frequently found in the Ashkenazi Jewish population. It's a rare genetic condition which is ultimately fatal. It's all caused by a deficiency of the aspartoacylase…
Emily Sadler is only 15. In March of 2017 she was diagnosed with thyroid cancer. This came as a huge shock to herself and loved ones, especially because thyroid cancer…
The government shutdown hasn't had any startling affects on rare disease research yet, but drug developers have voiced their concern that it could soon begin to hinder the research process.…
Happy Thursday! We hope everyone's week is going well. Today, we're highlighting a contribution from a cystinosis patient on the necessity of third-party medical providers. We're also sharing a story…
Xconomy.com has just released that funds have been raised for a variety of companies investigating therapies for rare conditions. Unfortunately, novel therapies and inventions cost million of dollars to develop. Thankfully,…
Myelodysplastic syndromes (MDS) is the name used to describe a group of stem cell disorders in which the body can't produce an adequate amount of platelets and blood cells. Currently,…
While medical students may go into the profession dwelling on the lives they can save, the inevitable fact of the profession is that there will be times when they…
According to an article recently published by Forbes, one data scientist wants to explain why data science has proven to be slow at identifying particular biomarkers in medical patients. Imran…
At age twenty one, Lisa was about to graduate college and had a promising future career in front of her. All that was left was graduation itself. The week before…
According to a story from PR Newswire, pancreatic cancer rates in the US are increasing by almost two percent each year. This puts the disease in position to become the…
According to a story from New Scientist, a team of scientists at the Institute of Cancer Research in London were able to develop an AI tool that was able to…
According to a story from BioPortfolio, the Bristol-Myers Squibb Company recently announced that the European Commission has recently approved the first combination treatment for renal cell carcinoma available in the…
A free MDS Patient & Family/Caregiver Forum, sponsored by The MDS Foundation Inc., will be held in Beverly Hills, California on February 2nd. When: Saturday, February 2nd, 2019 Where: Beverly…
According to a story from BioPortfolio, the keto-medical nutrition company Disruptive Nutrition recently announced that the first patient had been enrolled in a research study which will test a unique…
According to a story from Onclive, combination therapies are likely to play an increasing role in the treatment of chronic lymphocytic leukemia. In this video, leukemia treatment experts discuss the…
According to a story from The Conversation, scientists have determined that sex plays a significant role in outcomes when it comes to treating the rare and deadly brain cancer glioblastoma.…
The Right-To-Try Law is a federal legislation which allows terminally ill patients the opportunity to use experimental drugs that don't yet have FDA approval. It's controversial at best, but some…
Merkel cell carcinoma (MCC) is a form of skin cancer. In the United States, about 2,500 people are diagnosed with MCC each year. The condition is treated with chemotherapy, immune…
According to a press release published by Global News Wire, Australian pharmaceutical company Mesoblast Limited has completed recruitment for its phase 3 study of Revascor - their experimental drug to…
According to a story from Eurek Alert!, a team of scientists affiliated with the University of Helsinki have successfully developed a new, more useful Hirschsprung's disease mouse model. This disease…
According to a story from BioPortfolio, the pharmaceutical company Acer Therapeutics, Inc., recently announced the company's New Drug Application (NDA) has been accepted by the US Food and Drug Administration…
Every day the scientific community makes progress in the study of rare disease. Research in the field has advanced exponentially in recent years and we're starting to see more treatments…
According to a story from BioSpace, the biopharmaceutical company Liquidia Technologies, Inc., has recently released interim safety data for its open label Phase 3 clinical trial testing LIQ861. This data…
© Copyright 2024 Patient Worthy
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.
