Muscular Dystrophy Association’s Pompe Disease Patient Focused Drug Development Meeting Cleared by FDA

According to a story from, the Muscular Dystrophy Association (MDA) has recently announced the official approval of its Patient-Focused Drug Development (PFDD) meeting by the US Food and Drug Administration (FDA). This meeting will be focused on developing treatments for Pompe disease. The event is scheduled for March 9th, 2020 in Hyattsville, MD. The association has also partnered with the United Pompe Foundation (UPF) and the Acid Maltase Deficiency Association (AMDA) for the meeting.

About Pompe Disease

Pompe disease, which is also known as glycogen storage disease type II, is a genetic, metabolic disorder. This disease can cause damage to the nerves and muscles throughout the body and is the result of the excessive buildup of glycogen in the cellular lysosome. This occurs because of the deficiency of a certain enzyme. The disease is the result of a genetic mutation that appears on chromosome 17. Symptoms of Pompe disease vary depending on when it appears. They can include poor growth, trouble feeding, enlarged heart, poor muscle tone, muscle weakness, and breathing problems. There is also a late onset form that mostly differs by the absence of heart abnormalities. The primary treatment for Pompe disease is enzyme replacement. While this treatment can improve symptoms and survival, a high dosage is necessary and it primarily only halts disease progression. To learn more about Pompe disease, click here.

About Patient-Focused Drug Development Meetings

PFDD meetings are a critical opportunity that will allow for members of the Pompe disease patient community to voice their concerns to the FDA and other relevant parties on the subject of treating the illness. This will entail discussing the day to day challenges of living with the disease as well as articulating desired benefits of treatment and the potential risks that it can carry. The event will encourage regular audience feedback and participation and feature moderated panel discussions.

Current treatment options for Pompe disease are quite limited both in quantity and effectiveness. Patients will be able to discuss their experiences with the treatment approaches that are available and articulate what they hope future therapies will look like. 

The MDA plans to publish a report following the meeting called “Voice of the Patient” that will capture the content of the meeting and the perspectives that were shared by patients.


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