Every story has a turning point.
Real stories from real people living with late-onset Pompe disease (LOPD) who turned to POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat).
POMBILITI + OPFOLDA is a two-component therapy for adults with LOPD weighing 88 lbs or more who are not improving on their current enzyme replacement therapy (ERT).
Real stories from real people living with late-onset Pompe disease (LOPD) who turned to POMBILITI + OPFOLDA.
POMBILITI + OPFOLDA is a two-component therapy for adults with LOPD weighing 88 lbs or more who are not improving on their current enzyme replacement therapy (ERT).
The reality of LOPD
Everyone’s experience is different. Keeping track of how you’re doing on a daily basis can help you have more meaningful conversations with your doctor.
After all, some turning points are more obvious, like noticing difficulties with moving or breathing. Others can be more subtle, like noticing small changes that start to add up over time. If you find yourself noticing any changes, even subtle ones, turn to your doctor. Then, together, you can decide what’s best for you.
LOPD Turning Point Stories
Listen to real stories from real people living with LOPD who turned to POMBILITI + OPFOLDA.
Signs you may have reached a
turning point:
Doctors use the 6-Minute Walk Test and Forced Vital Capacity Test to help them keep track of how your LOPD is progressing. However, it’s always a good idea to monitor how you’re doing between visits. Noting any changes you’re experiencing, even small ones, can help you have more meaningful conversations with your doctor.
Realizing you’ve hit a plateau
LOPD progresses slowly, affecting each person’s muscle function in different ways. So, if you’ve noticed you’re not improving or are declining on your current ERT, it could be sign that you’ve reached a turning point and need to talk to your doctor.
“I noticed housework was becoming more difficult. Bending over and standing back up got harder. And I was over it.”
Kerri
Her StoryNoticing you’re declining on your current ERT
Realizing you’re not able to do the things you once could is a terrible feeling. If you’ve noticed you’re declining and it’s affecting your mobility or breathing, you may have reached a turning point. Consider making an appointment with your doctor to talk about what’s going on.
“First and foremost, be attuned to your body. Look for the subtle changes, whether it be breathing, walking, or balance.”
Scott
His StoryPerforming daily tasks and physical functions are more challenging
Does climbing stairs or doing daily tasks leave you exhausted? If you’re mentally nodding your head “yes,” this could be a sign it’s time to talk to your doctor about what’s going on. Together, you can discuss if you’ve reached your own turning point and next steps.
“On my previous treatments, I’d work on my car for only 20 to 30 minutes before I had to stop. Getting down low was extremely difficult, and I wasn’t able to get much done.”
Michael
His StoryTreatment effects are waning
If you feel like your current ERT isn’t providing the benefits it once did, you may have reached a turning point. Talk to your doctor about what you’re experiencing on your current ERT to get their advice on what to do next.
“Being 28 years old and seeing your body decline is really scary. So I needed to make a change.”
Veronica
Her StoryReady to make the turn?
If you notice yourself plateauing or declining in any way on your current treatment, it’s a good idea to have a heart-to-heart with your doctor. After all, the best advocate for your health is you.
Tips for talking to your doctor
Take regular notes
By taking regular notes and keeping track of how you’re feeling, you can give your care team a better picture of your experiences between visits.
Do your research
Research different treatments, like POMBILITI + OPFOLDA, and find out what they do, how they work, and what people on the treatment are saying. Then, discuss what you’ve learned with your doctor. Together, you can decide on next steps.
Bring a friend to your doctor visits
It’s easy to forget what was said at an appointment. Bringing a trusted friend or loved one along gives you another set of ears to help you remember what you and your doctor discussed.
Knowing when you’ve reached a turning point is the first step.
These resources will support you with the next one.
Be better prepared for talking to your doctor
To help you prepare for upcoming conversations with your doctor, we’ve made it easy for you to create and download your own discussion driver.
Create a Discussion Driver
Care at every step of your treatment journey
AMICUS ASSIST provides comprehensive support to people prescribed POMBILITI + OPFOLDA. A Patient Education Liaison (PEL) and a Case Manager are friendly faces standing by to help.
Connect With AMICUS ASSISTQuick links to helpful tools
Here’s a list of resources with information that could help you on your treatment journey.
Connect with an AMICUS ASSIST team member to learn more.
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