A recent study has demonstrated that neuromyelitis optica spectrum disorder (NMSOD) patients treated with eculizumab have a lower risk of adjudicated relapse compared to those treated with placebo. This study…
Incyte has withdrawn its New Drug Application (NDA) for parsaclisib, which is an investigational treatment for various types of lymphoma, including relapsed/refractory mantle cell lymphoma (MCL), follicular lymphoma, and marginal…
A new Phase 2 clinical trial will begin this year to explore NKT2152 (a HIF2a-inhibitor) in combination with Fotivda/Tivozanib (a VEGFR tyrosine kinase inhibitor) as a treatment for relapsed/refractory clear…
Those with recessive dystrophic epidermolysis bullosa (RDEB) have fragile, easily blistered skin. Unfortunately, because of dysfunctional tissue repair, wounds may take longer to heal – causing increased levels of scarring…
Targeted Oncology recently featured a discussion by Dr. Jennifer Brown, director of Dana Farber’s Chronic Lymphocytic Leukemia Center, on the interim results from the SEQUOIA Phase 3 trial. The trial…
KalVista Pharmaceuticals plans to initiate a Phase 3 trial for KVD900, a treatment for hereditary angioedema (HAE) after finding positive results from earlier Phase 1 and 2 trials. The Phase…
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…
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.
