Before you read on, make sure you take a look at Pt. 1 of our interview, which goes over what PGAP3 is, Lucy's story, and the reasons behind why Zach and Geri…
Zach and Geri Landman are devoted parents and physicians – a pain specialist and pediatrician, respectively – committed to making the world a better place for those around them. They…
According to a recent press release from Globe Newswire, Albireo Pharma announced that Lancet Gastroenterology published results of the PEDFIC 1 trial evaluating Bylvay® in the treatment of progressive…
A recent article published in Physicians Weekly and also by the National Institute of Health stated that immunosuppressive medication and kidney biopsies may not be necessary if children with…
In a news release from biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. ("Alnylam"), the company shared that positive data was available from the Phase 3 APOLLO-B clinical trial.…
In the U.S., the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. “Rare” refers to any condition affecting fewer…
According to a news release from biotechnology company Sound Pharmaceuticals, enrollment has begun in the Phase 3 STOPMD-3 clinical trial. So far, the first patient has been enrolled – with…
On July 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar for July. As always, these recurring meetings help provide critical updates to the rare disease community…
© 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.
