Orchard Therapeutics is investigating their gene therapy, OTL-102, for the treatment of X-linked chronic granulomatous disease (X-CGD). This treatment has recently received the Orphan Drug designation from the FDA, which…
According to a story from The ASCO Post, the promising results from a phase II clinical trial conducted last year suggests that CAR T-cell therapy could be an effective treatment…
Happy Thursday! This week, we're highlighting a story about a story about Karina, an EDS patient and advocate. Next, we have a story about a legal controversy that could affect…
According to a story from USA Today, 61 year old Jerry Devries is currently the oldest living patient with a very rare disease: Proteus syndrome. An unusual disease with only…
.jpg)
Janssen R&D, a division of Johnson & Johnson (J&J), has partnered with the UK Biobank and others according to a recent news release by 3BL Media. It is noteworthy…
According to a story from Pulmonary Advisor, the results of a recent analysis led researchers to conclude that systemic anticoagulation therapy can be beneficial for patients with idiopathic pulmonary arterial…
Hereditary Angioedema Hereditary angioedema (HAE) is a rare disease which causes swelling episodes throughout the body, including the airways, gastrointestinal system, and extremities. Most commonly, HAE is caused by a…
According to a story from Angioedema News, a team of researchers and scientists have successfully developed a tool that is able to measure disease activity in hereditary angioedema that is…
According to a story from the Stamford Advocate, rock legend Ozzy Osbourne is making plans to go on tour again next fall. This is despite the fact that he has…
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.
