A two-component therapy for adults with late-onset Pompe disease (LOPD) weighing 88 lbs or more who are not improving on their current enzyme replacement therapy (ERT).
SUPPORT + RESOURCES explore videos
EMMA
DIAGNOSED WITH LOPD IN 2009
EXPLORE VIDEOS ABOUT PEOPLE LIVING WITH LOPD
Learn how people manage their daily lives with LOPD and their therapy
EXPLORE VIDEOS ABOUT PEOPLE LIVING WITH LOPD
Learn how people manage their daily lives with LOPD and their therapy
Sean’s LOPD Story
Sean opens up about living with LOPD, the importance of not minimizing symptoms, and his experience with treatment.
Sandi’s LOPD Story
Sandi, a former nurse, talks about her diagnosis, treatment journey, and the importance of self-advocacy and setting small goals for herself.
Maddie’s LOPD story
Maddie details her diagnosis at age 11, her disease progression prior to switching to POMBILITI + OPFOLDA, and her experience since the switch.
Jared’s LOPD story
Jared discusses his 4-year diagnostic journey, his switch experience, and how his disease has helped him on his career path.
Emma’s LOPD story
Emma shares her diagnosis story, talks about the unique bond she shares with her sister, Maddie, and discusses her treatment goals.
Emma and Maddie share their experiences growing up with LOPD
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“Pompe has affected who I am. It started with wanting to know more about the disease. Now, I have my master’s degree in public health and work for the local health department.”
— Emma on how living with LOPD has inspired her throughout her life
