According to a story from Global News, Charleigh, the three year old daughter of Trevor Pollack and Jori Fales, was recently diagnosed with CLN2 Batten disease, which is a rare…
According to a story from Cancer Therapy Advisor, a recent study published in the journal Cancer Medicine indicated that chronic lymphocytic leukemia (CLL) patients who dealt with adverse events during treatment…
According to a press release from the French biotechnology company Inventiva, the company has completed recruitment for a phase 2 clinical trial evaluating its experimental mucopolysaccharidosis VI drug, odiparcil. About…
Von Willebrand disease is the most common inherited blood clotting disorder that affects humans. The disorder can also be acquired. Although it is considered common, the majority of cases do…
.png)
Social media, and especially Facebook, have become our “windows to the world”. According to a recent article that appeared in The Michigan Public Health News Center, scientists are using…
According to a story from Bioscience Today, amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease or motor neurone disease, has continued to be a mysterious disease for the…
According to a story from Charcot-Marie-Tooth News, a recent study was conducted that compared the presentation of symptoms between two different variants of Charcot-Marie-Tooth disease (CMT): CMT4B1 and CMT4B2. The…
.jpg)
The blood-brain barrier is a “gatekeeper” that prevents toxins and foreign matter in the bloodstream from traveling to the brain. Check Orphan recently carried an article that had been…
According to a publication from Pharmaceutical Technology, California-based Eiger Biopharmaceuticals is preparing to submit a New Drug Application (NDA) to the Food and Drug Administration (FDA) for their experimental progeria…
According to a publication from Stat, researchers from the University of Michigan-led Genes for Good program have recruited over 20,000 Americans through its smartphone app. Participants complete questionnaires on the…
According to a story from drugs.com, the pharmaceutical company Merck recently announced that the US Food and Drug Administration (FDA) has approved its drug pembrolizumab (marketed as Keytruda) as a…
Jane Biehl Jane Biehl has worked as a professor, rehabilitation counselor, and a librarian. She holds three different degrees, and spreads her knowledge through freelance writing. She also has a…
According to a story from marketscreener.com, the biotechnology company Roche recently announced the results from a company sponsored phase 3 clinical trial that compared the safety and efficacy of rituximab…
It is 2019, and minority populations still account for fewer than 10% of clinical trial participants. Two decades ago, the "NIH Policy and Guidelines on the Inclusion of Women and…
Eosinophilic Esophagitis (EoE) Eosinophilic esophagitis (EoE) is a rare and chronic disorder which affects the digestive system. Eosinophils are a type of white blood cell that help fight infections. This…
Chronic Kidney Disease Chronic kidney disease (CKD) is a rare condition which is defined as lasting and progressive kidney damage. The kidneys are responsible for filtering waste and fluids from…
Adapting to a long- term health condition, is not an easy road to be on. There are many phases we go through during our journey from denial to acceptance. It…
From Friday, June 7th to Sunday, June 9th, 2019, I had the privilege of attending the 2019 International Waldenstrom's Macroglobulinemia Foundation Educational Forum. This year, the annual event was held…
.jpg)
The California Institute for Regenerative Medicine (CIRM) has funded a study by a company called Forty Seven, Inc. The clinical trial is investigating the benefits of a drug therapy for two cancers…
According to a story from Global Genes, the drug developer SpringWorks Therapeutics announced that the US Food and Drug Administration (FDA) has granted the company Fast Track Designation for its…
Meet 29 year-old Cary Velazquez -- make-up artist in Puerto Rico with the rare disease osteogenesis imperfecta (OI), (also known as “brittle bone disease” and “glass bones disease”) who has…
IPain The International Pain Foundation (IPain) is a nonprofit organization dedicated to supporting those with chronic pain due to neurological, musculoskeletal, inflammatory, degenerative, and emotional conditions. They aim to improve…
According to a story from ucf.edu, a new partnership between Mayo Clinic and Limbitless Solutions is planning initiate a new clinical trial that aims to improve mobility options for patients…
According to a story from vtnews.vt.edu, a research team at Virginia Tech has made a critical discovery about the origins of Lyme arthritis, a painful and debilitating symptom that among…
Emily Ventura has never met in-person another person living with progressive familial intrahepatic cholestasis (PFIC), her daughter’s life-threatening ultra-rare genetic disease. That will change on June 21st when Emily and…
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.
