Pompe disease is a rare genetic disease characterized by symptoms such as muscle weakness, scoliosis, and heart problems. Dealing with these symptoms can be very stressful, and patients and caregivers usually need a lot of support.
Here are a few ways to find more information about Pompe disease and network with other patients, researchers, and clinicians.
Visit the websites of Pompe disease organizations
There are many nonprofit organizations that provide resources for Pompe disease. Some of these include:
- International Pompe Association
- Association for Glycogen Storage Disease
- Metabolic Support UK
- United Pompe Foundation
- Acid Maltase Deficiency Association
These websites offer ways to network with other patients, researchers, clinicians, and caregivers. The wealth of personal experiences shared on these sites provides opportunities to learn from others.
Subscribing to these sites gives you access to seminars, get-togethers, and first-hand information about upcoming clinical trials.
Check out resources by pharma companies
Pharmaceutical companies that develop specialized treatments offer dedicated portals containing information about the medicines they are developing and direct you to other resources and support pages.
For example, Sanofi Genzyme, makers of the enzyme replacement therapy (ERT) called Lumizyme, has a dedicated website for information about the therapy, safety information, tips on managing your health, support resources, and more. Sanofi also offers personalized patient services for help with disease education and assistance with health insurance.
Add your name to the Pompe disease registry
The Pompe disease registry (NCT00231400) is an international observation study sponsored by Sanofi Genzyme that tracks disease outcomes in Pompe disease patients. No experimental treatments are involved. Enrolling in the study can provide valuable information to researchers about the disease and help in the development of future therapies. You can ask your doctor to enroll you in the study if you have a confirmed diagnosis of Pompe disease.
Follow support groups on social media
Most Pompe disease organizations also have a presence on social media such as Facebook, YouTube, and Twitter. Follow them on these platforms to gain more insights, share personal experiences, and find local support groups in your area. They can also alert you of breaking developments in the field.
Subscribe to our website
Our website Pompe Disease News helps you stay abreast of new developments in Pompe disease research. It also offers health insights, information about various aspects of the disease, current treatment options, and upcoming therapies, as well as details about ongoing and upcoming clinical trials.
Subscribing to our newsletter can help you stay up to date with all the latest developments in the field of Pompe disease.
Last updated: Feb. 3, 2020
Pompe Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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