Propionic Acidemia Foundation
Dedicated to finding improved treatments and a cure for Propionic Acidemia.
08/06/2026
🍂 Got a story, update, or shoutout to share? We’re putting together our Fall Newsletter and would love to include your voice! 🧡
📬 : Send us your submissions by 9/1!
📧 : [email protected]
08/03/2026
Welcome, August! ☀️
What are your plans for this month?! Share back to school tips and pics!
🧡💛
08/03/2026
Congrats, Kate!
🎓 Join us in congratulating 2026 Guthrie-Koch Scholar Kate Lowry! 👏
A bassist, performer, and songwriter, Kate's passion for music has taken her to Berklee College of Music, where she begins her sophomore year this fall studying Bass Performance. She hopes to build a career as a touring and session musician.
"The Beatles were right—you really can get by with a little help from your friends."
🌟 Learn more about Kate and meet the other scholars: https://loom.ly/HzwRFG8
08/02/2026
PCCA is part of PaVe-GT. The learnings will help more than just the disorders within PaVe-GT. Thank you for sharing.
PaVe-GT's FDA Regulatory Review Experience with AAV Gene Therapy | Rodica Stan, PhD posted on the topic | LinkedIn I’m thrilled to share the PaVe-GT’s latest article published in Human Gene Therapy (open access) that describes our experience with the regulatory review at pre-IND and Type C meetings with the FDA for the program’s first AAV gene therapy candidate. Free to use templates and examples of regula...
08/02/2026
Living with PA ?
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. https://picnichealth.com/make-an-impact/compass
07/19/2026
First PA family get together in NYC. Everyone had a wonderful time and the food was so delicious.
We even had an improvised talent show from Nika!
Hoping to welcome more families next time.
07/17/2026
Congratulations to everyone. A special congrats to Kate. Keep making beautiful music.
🎉 Congratulations to flok's 2026 Guthrie-Koch Scholars!
Each year, the Guthrie-Koch Scholarship celebrates students living with inherited metabolic conditions who are pursuing ambitious educational goals alongside the demands of daily care.
Join us in congratulating this year's recipients:
Farah Baradei
Mitchell Magyar
Laiken Farris
Gabriela Palacios
Matthew Sheridan
Bryce Steach
Kate Lowry
Rosie Pallone
🌟 We'll be highlighting each scholar throughout the month!
👉 Read their inspiring stories: https://loom.ly/HzwRFG8
07/13/2026
Huge news! 🎉 The Propionic Acidemia Foundation is awarding a $50,000 research grant to Duke University’s Guofang Zhang — and it’s all thanks to YOUR support. Together, we’re pushing PA research forward. 💙
07/10/2026
Congrats! Great news. This will hopefully impact individuals with OA's including PA.
Children’s Hospital of Philadelphia Secures Five-year, up to $38.9 Million ARPA-H Award to Advance Personalized Gene Editing Therapies CHOP announced today that it has been awarded a five-year up to $38.9 million award from the Advanced Research Projects Agency for Health (ARPA H) THRIVE program to support the advancement of personalized gene therapy. Rebecca Ahrens-Nicklas, MD, PhD Children’s Hospital of Philadelphia (CHOP) anno...
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