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 announce two upcoming webinars for the Pyridoxine-Dependent Epilepsy (PDE) community.

Register for our 2024 Webinars for the PDE Community. After registering, you will receive a confirmation email containing information about joining the meeting.

Big news, everyone! We’re putting together the very first face-to-face meet-up for the PDE community.

Following the webinar, we identified several critical action items to better support families affected by PDE and advance our collective understanding and management of this condition.

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!