Janssen Pharmaceuticals has an ongoing Phase 1/2 clinical trial for the inherited retinal disease X-linked retinitis pigmentosa (XLRP). They are investigating an adeno-associated gene therapy called RPGR as a therapeutic…
Scares That Care Scares That Care is a 501(c)(3) organization, first established in 2007 by Joe Ripple, dedicated to fighting the "monsters" of childhood illnesses, breast cancer, and burns. They support…
The EveryLife Foundation has initiated a scholarship fund to help those with rare conditions pursue their academic dreams. These will be one-time awards of 5,000 dollars. Up to 32 patients will…
Biopharmaceutical company Theratechnologies Inc. recently announced that its therapy candidate tesamorelin is effective in the treatment of HIV-associated nonalcoholic steatohepatitis (NASH). According to new data, published in JCI Insight, tesamorelin…
In a recent webinar hosted by the International Waldenström's Macroglobulinemia Foundation (IWMF) and held on July 22nd, 2020, Dr. Jeffery Matous from the Colorado Blood Cancer Institute gave an in-depth…
BioMarin has recently submitted a Marketing Authorization Application to the European Medicines Agency for their achondroplasia treatment, vosoritide. As of now, the review of the application will begin in August.…
There's no misunderstanding this fact: renal-replacement therapy is effective for patients with severe acute kidney injuries (AKI). However, states Kenny Walter in MD Magazine, the efficacy of this treatment can depend…
According to a story from Charcot-Marie-Tooth News, a new nonprofit organization called Shark Tooth Cycling has recently begun operations. The mission of the organization is to encourage young people in…
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.
