La Jolla Pharmaceutical recently announced a new clinical trial targeting thalassemia. The trial, described as pivotal, investigates a drug referred to as LJPC-401. The treatment is intended to help patients…
According to a story from medicalxpress.com, a recent study suggests that cognitive deficits that can occur as side effects of targeted brain cancer drugs could be successfully reversed with therapy.…
According to a story from the National Institutes of Health, the innovative new CRISPR gene editing technology is being used in order to identify specific genes that could play a…
According to a story from drugs.com, kids and teenagers with chronic kidney disease (CKD) may develop problems with learning and cognition, a new study suggests. Researchers emphasize that patients on…
In 2016 the Centers for Disease Control verified that Zika virus caused birth defects such as microcephaly. This shocked the world. We witnessed the first mosquito-borne disease known to result…
Happy Friday, Patient Worthians! Today, we're highlighting a story about the donors who sacrifice their kidneys for rare patients, an update on CF research, and an exciting orphan drug designation.…
A new study has revealed several complications of a multiple sclerosis treatment. The studies illuminate difficulties surrounding a powerful drug treatment and potential pregnancies. Keep reading to learn more about the…
As an official Friend of Rare Disease Week, we are highlighting some of our heroes and rare disease warriors' stories. Below was submitted by the ADNP Kids Research Foundation and…
© Copyright 2024 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.
- 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.
