According to a press release from the Danish biopharmaceutical company Orphazyme, the Company has filled target enrollment for its phase 3 clinical study of arimoclomol in patients with amyotrophic lateral…
I had the pleasure of attending the Glut1 Deficiency Foundation’s 2019 Conference. While this article can't possibly do the event justice and encompass all it had to offer, we will…
According to a press release from American biotechnology company Celgene Corporation, the American Food and Drug Administration (FDA) recently approved the Company's drug Otezla (generic name apremilast) for the treatment…
Cyxone has just announced that the first person has been dosed in their Phase 1 clinical trial for Multiple Sclerosis (MS). This trial is investigating the effect of T20K. It…
No Cure, No Pay Emile Voest, an oncologist in the Netherlands, conceived the idea of a no cure, no pay model. After witnessing the exorbitant cost of drugs, some of…
According to a story from BioPortfolio, the biopharmaceutical company Genkyotex has recently announced that the US Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application.…
According to a press release from LifeMax Laboratories, Inc., the Food and Drug Administration (FDA) has granted the Company's experimental Netherton syndrome drug LM-030 (licensed from Novartis) Rare Pediatric Disease…
-150x150.jpg)
The Center for Disease Control (CDC) is urging doctors and researchers to investigate and to collect new data before the disease strikes again. An article in Ars Technica reports…
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.
