Rare Disease Week 2022 is taking place this year on February 22nd to March 2nd. Though normally held in Washington, DC, the EveryLife Foundation for Rare Diseases, which organizes the…
A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…
Rare Disease Day, which is recognized each year on the last day of February, is probably the biggest day for rare disease patient advocacy annually. There are always a wealth…
According to a study published in PLOS Medicine, only around half of pragmatic or 'real-world' trials collect data as reported by patients or involve patients as partners in research. This…
According to a story from Al Jazeera, the country of Panama is the only one in Central America with a law on the books that is specifically related to rare…
On January 27, 2022, The EveryLife Foundation for Rare Diseases, in partnership with the Biotechnology Innovation Organization (BIO), National Health Council, and Pharmaceutical Research and Manufacturers of America (PhRMA), hosted…
A study recently published in the American Journal of Transplantation evaluated whether or not transplanting the kidney of a pig could be a viable and safe options for human patients. This study…
For the last seven years, the Global Genes Rare Compassion Program has been providing medical students with an opportunity to bond with patients and families who have been diagnosed with…
Julia Lefelar, a resident of Gaithersburg, MD, was telling a friend about her years of seeing different doctors trying to find the cause of her illness. After a while, her…
The Critical Path Institute (C-Path), formed in 2005, is a nonprofit, independent (public/private) partnership. The Institute evolved from the FDA’s 2004 White Paper known as the Critical Path Report and…
On January 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted its first monthly webinar for 2022. In this month's program, the speakers provided an overview of the progress that…
According to a recent article, researchers were able to produce the first images of an enzyme that may figure out the unknown cause of a rare group of hereditary neurodegenerative…
This week the Guardian reported that a pig’s heart, which was genetically modified, had been transplanted into a patient by doctors who were trying to save his life. The patient was…
According to a recent article, Castle Creek Inc. has acquired Novavita Thera Inc. in an effort to expand its research and development efforts. Castle Creek Inc. Castle Creek Biosciences Inc.…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Newborn screening (NBS) is a public health service within the United States through which babies are screened for medical conditions that otherwise wouldn't be apparent at birth. Many of the…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Continued From Part One Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering.…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this status, “rare” conditions…
Kyleigh Kista, a 3 year old from Benonin South Africa needs heart surgery within the next 3 months to save her life. Kyleigh was diagnosed with tetralogy of Fallot with…
Pursuing an education can be difficult enough without any other extraneous factors. But what if you were also battling frequent infections, gastrointestinal problems, and recurrent hospitalizations? For Evan Brandon,…
The Myrovlytis Trust, a UK Charity focused on promoting research and advancing education for rare diseases, and Patient Worthy are now partners! This organization has focused primarily on Birt-Hogg-Dubé syndrome…
A new study has demonstrated that plasma desmosine could be an acute aortic syndrome biomarker. This also means it could be a potential diagnostic tool. The findings from this study…
One 66-year-old patient who lives in Poughkeepsie, N.Y. received the same diagnosis from three doctors. Each doctor suggested that his symptoms pointed to temporomandibular (TMJ) joint pain. The patient and…
After two years of in vitro fertilization treatments, Amber Freed, Age 38, and her husband Mark Freed of Denver, Colorado welcomed their twin babies Riley and Maxwell into the world.…
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