Oral GLP-1 Candidate Aleniglipron Shows Robust Weight Loss and Favorable Tolerability in Phase 2 Program
New Hope for Leigh Syndrome
Regulatory Fast-Track Transforms Lung Cancer Treatment Timeline; Gene Therapy Withdrawal Signals Market Challenges
LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
See the link ? Clicking there is the start to joining the stem cell registry. 40 years old or younger ? Your stem cells could BE The MATCH.
In rare disease and chronic illness communities, waiting isn’t just temporary — it can become part of everyday life.
Waiting for answers, for progress, for something to change.
It can feel like life is paused while everything around you keeps moving.
But your life is still valid in the in-between. Your pace is still enough.
If you’re living in uncertainty right now, we see you — and you’re not alone.
#RareDisease #RareDiseaseAwareness #ChronicIllness #ChronicIllnessAwareness #YouAreNotAlone #PatientWorthy
Today is International CHAMP1 Awareness Day 💙
CHAMP1-related neurodevelopmental disorder is rare, but the individuals and families impacted by it are strong, resilient, and deserving of understanding, support, and visibility.
Today we raise awareness, share knowledge, and stand with families worldwide as we work toward earlier diagnoses, better resources, and continued research.
💙 Every voice matters
💙 Awareness leads to understanding
💙 Together, we are stronger
#CHAMP1AwarenessDay #RareDiseaseAwareness #CHAMP1 #PatientWorthy
