This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Oscar was born in January 2020. He was a…
A new treatment for Rett syndrome is officially being studied in humans in Canada, meaning Canadian patients may have a new, viable treatment option in the future. Called TSHA-102, this…
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 think is of particular interest…
Rhabdomyosarcoma is a rare form of cancer that affects children. It was recently studied at St. Jude Children's Research Hospital, where a team of medical professionals examined cells that persist…
Dr. Omar P. Haqqani, MD FACS is currently the Chief of Vascular and Endovascular Surgery at the Vascular Health Clinics. He has a keen interest in understanding and addressing vascular…
A recent article shared the story of a woman diagnosed with cystinuria whose life was saved after her younger brother donated one of his kidneys. Cystinuria Cystinuria is a rare…
It’s finally May – and you know what that means! That’s right, this month is Huntington’s disease (HD) Awareness Month. The theme for this month’s HD Awareness Month is #LetsTalkAboutHD.…
Ultragenyx Pharmaceutical Inc. has just announced the first dosing in their Phase 2/3 trial investigating setrusumab (UX143) as a treatment for osteogenesis imperfecta (OI) patients. This trial is called Orbit,…
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.
