To the primary biliary cholangitis (PBC) community - it's time to rejoice! For the first time in nearly one decade, the U.S. Food and Drug administration (FDA) has approved a…
Parenthood is about raising the child you have, not the child you thought you'd have. Anthony and Lindsey Terling shared this message in their video "The Austin Way!" where the…
Tafenoquine is an anti-malarial drug that interferes with parasitic growth in the body. But could it be repurposed for use in another parasitic illness? That's what pharmaceutical company 60 Degrees…
Testing for rare conditions can be costly and time-consuming. In India, testing for spinal muscular atrophy (SMA), a rare genetic disorder, has traditionally cost between 3500 INR to 7000 INR…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Rare Disease Day may be long past in February, but the fight for rare disease awareness continues. Earlier this year, specialty pharmaceutical company Neuraxpharm Group ("Neuraxpharm") and biotechnology company Minoryx…
Effective disease management in inflammatory bowel disease (IBD), which encompasses Crohn’s disease and ulcerative colitis, is incredibly important. Managing your condition helps you maintain a better quality-of-life, reduces uncomfortable or…
Occasionally the acronym NEGU may appear in a social media post. It has been established as a rallying cry and support for people with cancer or other debilitating diseases. It…
If you have read my books or articles, you have probably noticed that I talk about self-care quite a bit. I think self-care is extremely important for anyone, whether they…
BioSpace recently published a press release discussing the interim results highlighting a Coya Therapeutics’ study. The study evaluates the safety, tolerability, and biological activity of LD IL-2 in 38 patients…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Kari Rosbeck, the…
Jianmin Fang, M.D. the CEO of the biotechnology company RemeGen, recently announced in PRNewswire that the company is continuing its significant progress in its research of Telitacicept (RC18) to treat…
An estimated 25% of people with Clostridioides difficile (C. diff) infections experience recurrence following successful antibiotic intervention. After three or more recurrent infections, the risk of developing another infection sits…
People with idiopathic pulmonary fibrosis (IPF) already have two FDA-approved treatment options: Ofev (nintedanib) and Esbriet (pirfenidone). But biotech company Agomab Therapeutics ("Agomab") is working to introduce another potential therapeutic…
Written by Mike Dobbyn “Life expectancy: Early to late teens.” It was March 20th, 2019, and I was reading my son’s obituary on my iPhone while sitting in the geneticist’s…
You might have heard of Orphan Drug designation - and maybe even Rare Pediatric Disease designation. But have you heard of Regenerative Medicine Advanced Therapy (RMAT) designation? Described in Section…
Up to 10% of heart failure cases could be caused by cardiac amyloidosis. Yet amyloidosis remains underdiagnosed and misunderstood. Broadening our understanding of this rare disease could contribute to earlier…
Written by Judith Hill, Communications and Events Director, National Alliance for Eye and Vision Research In a remarkable show of unity and commitment to eye health, the Alliance for Eye…
The Phase 1b/2a RESOLVE clinical study set out to identify how safe and effective EP-104GI is for people living with a chronic immune-mediated disease called eosinophilic esophagitis (EoE). People with…
In March 2024, Mayotte—a department of France, or a territory considered part of France—health authorities shared that the island was dealing with an outbreak of cholera. Cholera is an acute…
A 2008 report published in Current Psychiatry Reports found that approximately 1% of people with schizophrenia also have a rare genetic disorder called 22q11.2 deletion syndrome. This suggests that schizophrenia…
On May 17, 2024, Ohio was graced with a unique, Wizard of Oz-themed fundraising event called "An Event to End NF." NF, in this case, stands for neurofibromatosis, a rare genetic…
Just about one month ago, clinical-stage biotechnology company AnaptysBio announced the availability of positive top-line results from two Phase 3 clinical studies: GEMINI-1 and GEMINI-2. Both studies sought to assess…
Researchers recently linked Cushing syndrome remission to the development of de novo (first occurrence) diseases, including autoimmune diseases, in various patients. But how did they reach this point? The 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.
