The World Federation of Hemophilia's Virtual Summit: Connecting the Global Bleeding Disorders Community June 15-19, 2020 In light of the cancellation of WFH's World Summit due to the ongoing coronavirus/COVID-19…
By Danielle Bradshaw from In The Cloud Copy Expressive language sampling, or ELS, could potentially help researchers better measure how well treatments work for patients with fragile X syndrome or…
Nicox SA, an international ophthalmology company, recently announced a Phase 3 clinical trial for NCX 470 to lower intraocular eye pressure. The article recently appeared in Eye Wire News. The…
Happy Friday! The world is full of difficult news right now. If you're looking for ways to educate yourself on some of the problems facing the world right now, especially…
An estimated 25-30 million Americans are part of the rare disease community. If you're one of them, you share a common (yet uncommon) experience: public scrutiny, difficulties in diagnoses, treatment…
June is recognized as Myasthenia Gravis Awareness Month. Primarily organized by the Myasthenia Gravis Foundation of America (MGFA), this event is focused on spreading awareness as well as boosting efforts…
According to a story from PR Newswire, CureDuchenne Ventures has recently announced the initiation of its CureDuchenne Ventures 2020 Pitch Contest. This marks the first time that the contest has…
According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals has recently announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its…
According to a story from BioSpace, the biopharmaceutical company MacroGenics, Inc. has just announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's…
Acadia Pharmaceuticals recently released the results of their Phase 3 trial of Nuplazid, a treatment for the psychosis that Parkinson's disease patients experience. This medication has been approved since 2016,…
Gut bacteria has been connected to a number of conditions: depression, diabetes, and now Parkinson's disease. A study published in Annals of Neurology has shown that gut bacteria is linked…
The RARE Drug Development Symposium June 11-12, 2020 NOTE: Due to the ongoing coronavirus/COVID-19 pandemic, this event is now virtual. The RARE Drug Development Symposium is intended as an event…
According to Disability Horizons, an extremely talented filmmaker with muscular dystrophy recently won an Award of Recognition from The Best Shorts Film Competition. Stephanie Castelete-Tyrrell's film, entitled Muscle Warriors, documents…
PharmaEssentia Corporation, a biopharmaceutical company located in Burlington, Massachusetts, has recently seen their Biologics License Application (BLA) approved by the FDA. This application was submitted for ropeginterferon alfa-2b, a treatment…
Exercising has many physical and emotional benefits. It can manage blood sugar levels, improve your mood, assist with weight loss, or even strengthen your memory. Endurance training, specifically, benefits tissue…
According to a story from PR Newswire, the National Organization for Rare Disorders (NORD) has recently awarded a total of eleven new grant awards that will help fund essential research…
According to a story from EurekAlert!, a recent international study is utilizing data from 45 patients to describe a mostly unknown new rare genetic disorder. The researchers are recommending that…
Patient registries are a great way for patients to get involved in shaping future research around their conditions. Many times, getting involved in a rare disease (or rare disorder)…
According to a story from Sarcoidosis News, while the global challenge of the COVID-19 pandemic has been an obstacle for advancement in many rare diseases with trials and research grinding…
As reported in GSK, the FDA has just granted priority review to Nucala (mepolizumab), a targeted biological treatment to help patients with hypereosinophilic syndrome (HES). If the medication is accepted,…
Ultomiris, a product of Alexion Pharmaceuticals, has recently been studied in a Phase 3 clinical trial. This study was intended to ultomiris' effect on life-threatening complications of atypical hemolytic uremic…
According to a story from Neurology Advisor, a recent study has found that patients living with neuromyelitis optica spectrum disorder (NMOSD) are at an increased risk of experiencing bone loss,…
Kimberly Underhay Kimberly Underhay is a Canadian who has survived cancer. She has also been diagnosed with osteoarthritis and has to use a walker. Her disabilities have left her unable…
Neurofibromatosis Neurofibromatosis is a rare condition that presents in different forms. Neurofibromatosis type one (NF1) causes tumors to grow on the plexiform neurofibromas, a type of nerve in the body. These tumors…
In a recent article that appeared in Science Magazine, Amy Boland described her twelve-year quest to find a cure for the cancer that invaded her lymph system. At first,…
© 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.
