Understanding Pyridoxine-Dependent Epilepsy and Dietary Management: A Guide for Families.

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.

Upcoming Webinars in April and May for the PDE Community.

Hi there, You are invited to a Zoom meeting.When: Mar 28, 2024 12:00 PM Eastern Time (US and Canada) Register in advance for this meeting:https://zoom.us/meeting/register/tJIodO2sqD0iGNC8Xq5xZKr0O8pSPQnck0-A 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.