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: