Discussion on Right to Try legislation continues, and the federal government could soon change existing rules. Many people wonder whether or not patients should have the right to try potentially…
While learning about cancer at a molecular level while being a medical student, Omar Salman from Virginia Tech Carillon School of Medicine learned much more firsthand when he was diagnosed…
According to a story from asianscientist.com, a group of Japanese researchers have managed to unearth a genetic mutation that is associated with progressive supranuclear palsy. The discovery is helping scientists…
The EveryLife Foundation for Rare Diseases hosted a panel discussion on Right to Try Proposals, as part of their 2018 Legislative Program for Rare Disease Week on Capitol Hill. What…
According to a story from triblive.com, Roger Ziegler has self-published a book about his family's experience with his son Noah. Noah is now a healthy 24-year-old, but there were multiple…
According to a story from the Lancashire Telegraph, Dr. John Timms, age 48, was recently diagnosed with a rare and deadly form of cancer: mesothelioma. Dr. Timms had committed much…
A gene therapy for Pompe disease is the first treatment of its kind to be clinically tested, reports Duke University. Researchers at the University, working with NCATS Therapeutics for Rare…
One of the greatest gifts of being a kid is imagination. When you're a kid, anything is possible and even the hardest times can turn into something so much better…
For so many in the rare disease community, courage isn't a just a virtue - it's a necessity. Many have to overcome odds and obstacles that most others don't have…
According to a story from curetoday.com, a recent study indicated that many patients with uveal melanoma reported having unmet psychological and informational needs, particularly in the first several months after…
The average American might not be interested in walking two miles every day. However, for Patricia Coleman, it is an activity in which she proudly partakes! In honor of Sarcoidosis…
Ashley Moore survived cancer. For many people, that’s enough. Moore, however, chose to give back in a surprisingly direct and immediate way. She decided to become a nurse. Keep reading…
We hope that everybody is having a great Easter, Passover, or first taste of spring! This week we have a story about sisters with Batten disease who are finally being…
As we close out the month of March - it's important to give a little thought to some of the lesser-known rare diseases that are often overlooked. Bleeding disorders is…
According to a story from cancercompass.com, a recent study has demonstrated that income level is a significant factor in determining survival for patients with anal cancer. In general, the study…
According to a story from GlobeNewswire, the biotechnology company argenx announced recently that its investigational therapy ARGX-113 was granted orphan drug status by the European Commission. This designation is based…
According to an article from the Augusta Free Press, a research team from Vanderbilt University and the Virginia Tech Carilion Research Institute has successfully managed to uncover the manner in…
According to a story from mysunshinecoast.com.au, an international research team has uncovered a potential biomarker that could help develop treatments for multiple myeloma, a rare form of cancer. Currently, multiple…
According to a story from the Nottingham Post, the family members of brain cancer patients are putting their time and money towards the funding of a cutting edge MRI scanner…
According to a story from ibdnewstoday.com, a recent study from Italy suggests that people with severe cases of inflammatory bowel disease are more likely to develop non-alcoholic fatty liver disease.…
Michael Mullan was only a baby when he was diagnosed with cancer for the first time-- specifically neuroblastoma, a childhood cancer which had a low survival rate. Since then, he…
One of Patient Worthy’s partners, The MDS Foundation, will be hosting another FREE event! Event Summary: Whether you are a newly diagnosed patient, a long-term survivor, or a caregiver this event…
A recently published study examines the response of motor nerve cells to motor neuron disease. Researchers hope that by more completely understanding this cellular response that they may be able…
According to a story from Everyday Health, The U.S. Food and Drug Administration (FDA), has recently approved a new drug for the treatment of recently diagnosed patients with Hodgkin disease.…
According to a story from GlobeNewswire, The pharmaceutical company AveXis, Inc., a company that specializes in the development of gene therapies, recently announced that its candidate AVXS-101 was awarded SAKIGAKE…
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