Propionic Acidemia Foundation

06/14/2026

🧬 New Clinical Trial for Propionic Acidemia (PCCA):

A new Phase 1 clinical trial has been registered to study AAVrh10-PCCA, an investigational gene therapy for children with propionic acidemia caused by PCCA mutations. This study is currently enrolling children 6 months to 2 years of age.

🔗 Learn more: https://clinicaltrials.gov/study/NCT07643844

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06/11/2026

Propionic Acidemia Foundation is excited to share an opportunity to participate in the COMPASS Study, sponsored by Moderna and supported by PicnicHealth. This observational study is designed to better understand real-life experiences of individuals living with methylmalonic acidemia (MMA) due to mutase deficiency (MUT) and propionic acidemia (PA), beyond what is captured in clinical trials.
● No new treatments required.
● Your or your loved one’s medical records are collected securely.
● $250 + free access to all your organized health records as compensation for your time.
Your participation could help improve care for everyone living with PA.
Sign up today. https://go.pic.nc/1ov

06/01/2026

Living wit PA ? Your voice matters.
The COMPASS Study is an observational research effort exploring what life is really like for
people in our community.
✨ No new treatments required
✨ Your or your loved one’s medical records are collected securely
✨ $250 + free access to your or your loved one’s consolidated medical records as
compensation for your time
By participating, you can help researchers better understand PA and shape the future of care
for everyone affected.
Learn more and see if you are eligible.

Help advance research in methylmalonic acidemia (MMA) and propionic acidemia (PA) from home. Complete surveys, get $250, and access your medical records.

06/01/2026

🌊 Let June be filled with moments that feel like sunshine ☀️

05/29/2026

Loss of pyruvate carboxylase suppresses lethality in propionic acidemia

Check out this new article. Thank you to all of our families, researchers and donors for continuing to move research forward. Your donations help fund valuable research that increases the knowledge on propionic acidemia in order to help find new treatments and bring hope to families. https://www.cell.com/cell-reports/fulltext/S2211-1247(26)00435-3

05/25/2026

Remembering the brave and honoring their sacrifice. ❤️

05/22/2026

Great news! MMA research and trials will help us gain new knowledge and brings us closer to PA gene therapy.

MMA-101, an NIH-supported gene therapy for a rare metabolic disorder, has received FDA clearance to begin clinical testing.

05/18/2026

Learning, sharing, networking….

05/18/2026

Jill Chertow is at SIMD in Puerto Rico. Stop by the Propionic Acidemia Foundation Booth!

05/10/2026

Happy Mother’s Day to every PA mom who loves fiercely, advocates endlessly, and keeps going even on the hardest days. Your strength is felt, your love is seen, and today we’re celebrating each of you with so much gratitude. ❤️💐