Great news for those affected by rare metabolic disorders! Moderna’s new investigational treatment for methylmalonic acidemia (MMA), and work with Propionic Acidemia. This initiative aims to accelerate the development of promising therapies for rare diseases.

Dr. Houten’s work on substrate reduction therapy for GA1 and its implications for related pathways such as PDE offers a glimpse into the future of treatment for rare genetic disorders.

We are thrilled to extend a warm invitation to all of you for an upcoming Zoom meeting designed to bring us closer together.

We’re excited to share a unique opportunity for you to contribute directly to the future of PDE research.

Big news – we’re hosting our FIRST webinar on Pyridoxine-Dependent Epilepsy (PDE) research on January 25th, and you’re invited!

This blog post introduces the CHARLIE Consortium, a group of researchers dedicated to studying Pyridoxine-Dependent Epilepsy (PDE).