According to a story from BioPortfolio, the biotechnology company Tiziana Life Sciences plc has announced the release of preliminary topline data findings from its phase 2a clinical trial, which tested…
In a time where we get bombarded by all sorts of troubling news -- it's so soul-refreshing to see these truly inspirational moments go viral. A video of limb different,…
In an exclusive "Good Morning America" interview this week, Dr. Priscilla Chan announced the launch of Rare As One, a new program from the Chan Zuckerberg Initiative where she is co-CEO…
According to a story from Pulmonary Arterial Hypertension News, data from an open label extension trial of the drug Orenitram as a treatment for pulmonary arterial hypertension has demonstrated that…
.jpg)
The number of people in the U.S. who are permanently disabled or who die each year as a result of misdiagnosis is estimated to be over 100,000. EurekAlert recently reported…
TGIF! Unless you're on vacation in which case, every day is *basically* Friday. This week, we're bringing four articles to read as you escape the summer heat. We have a…
According to a story from Verdict Medical Devices, CVS Health has recently initiated a clinical trial which will test a device called HemoCare. This device is being developed by DEKA…
According to a story from Fabry Disease News, a recent study has found that despite changes to sperm cells, the fertility of men with Fabry disease is not affected. The…
The Immunization Action Coalition (IAC) Express Newsletter reports that 981 cases of measles have been reported so far this year, with 41 new cases added just last week. Rare disease kids…
According to a story from BioSpace, CTI BioPharma Corp. has recently concluded an End of Phase 2A meeting with representatives from the US Food and Drug Administration (FDA). This meeting…
The Sleeping Bear Dunes National Lakeshore Park, located in Michigan, has launched an innovated program to make their park more accessible to those living with a disability. They call it…
According to a story from BioPortfolio, the biopharmaceutical company CStone Pharmaceuticals has recently announced that China's National Medical Product Administration (NMPA) has issued approval for the company to conduct a…
.jpg)
Many patients may not be aware of the vital role they play in medical discoveries, or that without their participation there would be no medical discoveries. CheckOrphan recently presented…
According to a story from The Washington Post, two families affected by the rare disease spinal muscular atrophy have cause for celebration after UnitedHealthcare agreed to cover their treatment with…
.jpg)
Genomic studies and advances in technology have increased researchers’ knowledge of several genetic syndromes that put people at risk for acute myeloid leukemia (AML). An article that appeared in…
Last week, the New England Journal of Medicine published a report about a rare, disease-like infection that is a real cautionary tale. A 41-year-old British woman was diagnosed with acanthamoeba keratitis…
As recently reported in CheckOrphan, a new drug for sickle cell disease has quickly ascended through the FDA approval processes in an accelerated review. A New Sickle Cell Drug Shrouded…
Some promising news from across the pond! A new international consortium funded by the European Union will focus on speeding up the diagnosis and development of rare diseases and treatments.…
Vertex Pharmaceuticals announced the submission of a New Drug Application (NDA) to the FDA for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen for treating Cystic Fibrosis (CF). This…
The Mental Health Rare Genetic Disease Network (MHRGDN) is a newly launched system, developed by the National Institute of Mental Health. The MHRGDN spans 15 research sites across the country…
Learning that you or a loved one has a rare genetic disease can be scary. Doctors often are not very well versed in these types of disorders and may require…
Cyxone has just announced that the first person has been dosed in their Phase 1 clinical trial for Multiple Sclerosis (MS). This trial is investigating the effect of T20K. It…
According to a story from BioPortfolio, the company Novocure has recently announced that Medicare has released final local coverage determinations for its product Optune. Optune is used to treat patients…
No Cure, No Pay Emile Voest, an oncologist in the Netherlands, conceived the idea of a no cure, no pay model. After witnessing the exorbitant cost of drugs, some of…
Throughout my daughter Kelley’s 45-year life with a rare disease, there were numerous times that we needed to either call 911 or take her to the Emergency Department at a…
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.
