As everyone knows, due to the ongoing COVID-19 pandemic, the Patient-Focused Drug Development Meeting (PFDD) that was originally scheduled for March 09, 2020, was postponed.

It has been moved to a Virtual Meeting that will take place Monday, July 13, 2020, from 12:00-4:00 EST.

This is a wonderful opportunity for as many people in our as possible to participate and to make our voices heard. Attendees will include representatives from the Food and Drug Administration (FDA) and industry.

For more information on how you can participate, please click here.

For more information on the meeting, including as Agenda, please click here.

Remember, Together We Are Strong! Let’s make our voices heard on Monday!


The AMDA is excited to announce that it is partnering with the MDA to host a Patient Focused Drug Development Meeting (PFDD) on March 09, 2020.

The PFDD meeting is an opportunity for patients and families living with Pompe disease to share their perspective and inform the FDA and other key stakeholders about their experiences living with Pompe disease and how they view the potential benefits and risks of treatments for Pompe. This input will help inform FDA’s regulatory decision making and oversight during therapy development and in the regulatory review process.

It is a wonderful opportunity for our voices to be heard and the AMDA encourages everyone to participate if they are able.

For more information on this exciting opportunity, please read the MDA’s press release here.

More information can also be found on the following website: