Rare Classroom: Stevens-Johnson Syndrome
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
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
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD.
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we
In March 2023, 14-year-old Rylie Erbacher visited Washington, D.C. for a one-of-a-kind experience. The teen, who has spinal muscular atrophy (SMA) type 2, was one
Have you ever heard of the Orphan Drug designation? This designation, created through the Orphan Drug Act and granted by the U.S. Food and Drug
Rinvoq (upadacitinib) is a prescription JAK inhibitor that works by blocking pro-inflammatory signaling within cells. In the past, Rinvoq was approved for indications like rheumatoid
For patients who were given a diagnosis of advanced, inoperable (unresectable) biliary tract cancer, an article appeared in Healio with some encouraging news.
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA’s nod moves
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we
Republished with permission from Personalize My Medicine, a Patient Worthy partner organization. Written by Eleanor Doherty Glossary Antibody, Antidepressant, Antigen, Apoptosis, Astrocyte, CAR-T Cell Therapy, Atypia, Clinical Trial, CNS, Dendritic Cell Vaccine, Epidermal Growth Factor, Ex-Vivo, Glioblastoma
The National Organization for Rare Disorders (NORD), a Patient Worthy partner, formed in 1983 to provide support and education to people and families living with
On May 10, 2023, reports EMPR, the U.S. FDA approved Elfabrio (pegunigalsidase alfa-iwxj) for adults who are living with Fabry disease. Developed by Protalix Biotherapeutics
In 2014, head coach Jimbo Fisher led the Florida State Seminoles to win the BCS National Championship Game. He’s now head coach of the Texas
© Copyright 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.
What best describes you when it comes to rare disease? (check all that apply)
- 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.