Breaking News in Pompe Disease Treatment

Celebrating the Approval of Pombiliti + Opfolda for Treatment of Pompe Disease [Muscular Dystrophy Association]( [Breaking News in Pompe Disease Treatment ]( MDA Celebrates the Approval of Pombiliti + Opfolda for Treatment of Pompe Disease The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of Pombiliti™ + Opfolda™ for adults living with late-onset [Pompe disease](. "We are extremely pleased with the recent approval of Pombiliti and Opfolda,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. “MDA funded the foundational work at Duke University that contributed to the development of the first drug approved for Pompe, Myozyme, and it’s gratifying to see the evolution of new therapies. MDA will continue to stand by the Pompe community in support of these efforts.” Pombiliti + Opfolda is a groundbreaking step in advancing care for Pompe disease, also known as acid maltase deficiency. This new treatment is a novel two-component therapy that will be available for qualified adults who are not improving on their current enzyme replacement therapy (ERT) [Read the Full Announcement]( Investing in Pompe Disease Research Since Day One Since its inception, MDA has invested more than $5 million in Pompe disease research, continuing a commitment to fund the advancement of the understanding and treatment of [metabolic muscle disorders]( like Pompe disease. Until recently, there was no treatment for Pompe disease, and the only remedy was supportive medical care. In 2006, the FDA granted approval for the use of Myozyme as a treatment for infantile-onset Pompe disease, a drug that was developed by Genzyme Corp. of Cambridge, Mass., with initial funding support from MDA. Pombiliti + Opfolda will join a new legacy of groundbreaking treatments for Pompe disease, supported by MDA from day one. [Learn More]( Help Turn Research into Reality “The approval of Pombiliti and Opfolda provides another option for patients with Pompe disease,” said Paul Melmeyer, Vice President, Public Policy and Advocacy, MDA. “We are grateful to the Pompe community who have helped advance this therapy...Pombiliti and Opfolda has the potential to become the next standard of care in Pompe disease and should make a true difference in the lives of patients and their families.” Celebrating this groundbreaking treatment is possible because of dedicated donors who help make treatments like Pombiliti + Opfolda a reality for many who live with rare neuromuscular diseases like Pompe disease. Consider [giving a gift]( to help MDA continue these advancements for years to come. --------------------------------------------------------------- [Facebook]( [Twitter]( [Instagram]( [LinkedIn]( [YouTube]( [MDA.org]( © 2023 Muscular Dystrophy Association, Inc.. All rights reserved. 1016 W Jackson Blvd #1073 Chicago, IL 60607 1-800-572-1717 [Unsubscribe]( | [State Fundraising Notices]( The Muscular Dystrophy Association (MDA) is a qualified 501(c)(3) tax-exempt organization. [View Online]( | [Subscription Center]( | [Privacy Policy](