Propionic Acidemia Foundation

🧬 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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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

go.pic.nc

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