Welcome to the International Pompe Association (IPA)
The International Pompe Association (IPA) is excited about our plan to host an international fundraiser for the Second Annual International Pompe Day (April 15, 2015). And we need YOUR help!
As with selecting the date, logo, and slogan for International Pompe Day, the IPA would like to ask the international Pompe community for its help in choosing a fundraiser “theme". Most people are aware of the extreme success of the ALS Ice Bucket Challenge. What made it so successful is that it: (1) was easy for people to do; (2) it was more or less free (just needed a bucket, ice, and water); and (3) it was fun for others to watch online! We have received five (5) terrific suggestions for potential fundraisers and now we need YOUR help in choosing OUR fundraiser!
Please visit the International Pompe Day website and cast your vote today! Vote for the fundraiser suggestion that you can't wait to be a part of!
Abstracts from the 7th European Symposium on Steps Forward in Pompe Disease (Turin, Italy, November 21–22, 2014) are published in the Journal of Neuromuscular Diseases and now available.
Topics included are:
- Late-Onset Pompe Disease: A Multisystemic Disorder
- Early Recognition of Late-Onset Pompe Disease by Skeletal Muscle Signs and Symptoms
- Early Recognition of Late-Onset Pompe Disease by Respiratory Muscle Signs and Symptoms
- Eight years of Experience with Alglucosidase Alpha Treatment: Facts and Perspectives
- Emerging Role of Muscle Imaging in Diagnosis and Evaluation of Pompe Disease Patients
- Best Standards of Care for Patients with Late-Onset Pompe Disease – A Canadian Perspective
- and others
Read more in the Journal of Neuromuscular Diseases, Volume 2, Supplement 1/2015
Issue Title: Abstracts from the 7th European Symposium on Steps Forward in Pompe Disease: Turin, Itally, November 21 – 22, 2014
(Text by Maryze)
As of the 1st of February 2015, the Australian government has approved enzyme replacement therapy (ERT) for young Australians with Pompe disease. For the age group of 2 - 18 years ERT is now on the list of the Life Saving Drugs Program. In 2010 the ERT was already approved for infants with Pompe disease under the age of 2 years.
The Australian Pompe Association (APA) has worked hard and for many years to achieve this success. In the online newspaper The Age Federal Politics an article can be read on the impact of this achievement.
- Australian Pompe Association - Children given lifeline
- Minister of Health - Life-saving drug for young Pompe patients
- Genzyme - Young Australians with Pompe Disease now able to access treatment via the Life Saving Drugs Program
Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.