Spinal muscular atrophy (SMA) patients aged two months and older will now have an at-home treatment option in Europe. The European Commission (EC) has approved of Evrysdi for those who…
As of November 20, Zokinvy has been approved by the FDA for the treatment of Hutchinson-Gilford progeria syndrome (progeria) and a few other progeroid laminopathies in patients aged one year…
New medicines for Parkinson’s may be around the corner. The United States Food and Drug Administration (US FDA) recently approved one drug, and accepted application another. Both new drugs treat…
Earlier this month, the FDA approved Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. The final results indicated a response rate of 54.5%. This is the first FDA-approved treatment…
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.
