In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Where do I begin? Thank you…
Happy Friday! September is a packed with rare disease awareness, as we've been featuring all month long. We wanted to make sure to draw attention to Dystonia Awareness Month, which…
In a recent press release, precision therapy company Blueprint Medicines Corporation announced positive results from two clinical trials: EXPLORER and PATHFINDER. The trials examined the safety, efficacy, and tolerability…
Knowing how to help treat your disease and being able to afford those needs are two different questions. As reported in Newswise, in a study from Harvey L. Neiman Health Policy Institute, researchers…
As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…
CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…
by Lauren Taylor from In The Cloud Copy Rheumatoid arthritis (RA) is an auto-immune condition that occurs when the body’s immune system mistakenly targets the joint linings causing inflammation, pain,…
According to a story from Charcot-Marie-Tooth News, an experimental HDAC6 inhibitor called CKD-504 appeared to restore myelination in mouse and cell models of Charcot-Marie-Tooth disease type 1A in a recent…
Dr. Naveen Pemmaraju recently interviewed with Targeted Oncology and discussed the many advances in myeloproliferative neoplasms (MPNs) treatment as well as the role of social media during the pandemic.…
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.
