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
Link: http://iospress.metapress.com/content/qm2651450586

(Text by Maryze)

As of the 1^st 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.

Press Releases

• 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.

Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLDSymposium(TM) 2015

• Optimized for Efficient Targeting to Multiple Key Muscle Tissues of Disease
• Significantly Better Than Approved ERT in Reducing Disease Substrate (Glycogen)
• Addition of Chaperone Further Improves Glycogen Reduction

ORLANDO, Fla. and CRANBURY, N.J., Feb. 10, 2015 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today highlighted results from preclinical studies of its next-generation Pompe ERT at WORLDSymposium™ 2015 in Orlando, Florida.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "Our goal is to develop a fundamentally better therapy for patients living with Pompe disease. The results presented at WORLDSymposium suggest that we have made significant progress with our Pompe program as we look toward addressing some of the major challenges with the current approved therapy including enzyme activity and stability; targeting and uptake; and tolerability and immunogenicity. These results also validate our internal biologics capabilities to develop and manufacture our own cell line at clinical scale. We look forward to moving our next-generation ERT into the clinic later this year." ...

Read more

• Press release (external link)
• Press release (PDF, 21 kB)

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.