
FDA Places Clinical Holds on REGENXBIO’s MPS Gene Therapy Programs After Tumor Detected in Trial Participant
Austin Newman - February 6, 2026LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
Meet Amber! She was diagnosed with Granulomatosis with Polyangiitis (GPA) in 2017. Through traditional medicine, holistic care, and fierce self-advocacy, Amber is now in remission! She shares her story to educate medical professionals and empower others living with GPA. For Rare Disease Month, her message is clear: we are not alone. Together we share our stories and learn from each other, and knowledge is power! Thanks Honey I Roam for sharing your story with us!
#granulomatosiswithpolyangitis #Wegeners #rarediseasemonth #RareDisease #rarebutnotalone #WeCareAboutRare #SHAREYOURSTRIPES #shareyourstory #PatientWorthy
... See MoreSee Less
- likes love 4
- Shares: 0
- Comments: 1
Mississippi Metabolics Foundation #RareDiseaseThis is a monumental day for rare disease patients!!! All our hard work has been realized. ... See MoreSee Less

Let's talk about it: What's the most difficult part of explaining your rare disease to people who don't understand? Share in the comments! 💙 #rarediseasemonth #rarediseaseawareness #youarenotalone ... See MoreSee Less



