CRANBURY, N.J., May 15, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) today announced positive functional data from initial patients in a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease. Patients who completed six months of treatment with ATB200/AT2221 showed improvements in the six-minute walk test (6MWT) distance and other measures of motor function, in addition to stability or improvements in forced vital capacity (FVC). Consistent with previous results presented at the 2017 WORLDSymposium™, patients treated with ATB200/AT2221 continue to show improvements in biomarkers of muscle damage and disease substrate.

"We are very pleased to see improvements in six minute walk distance and other measures of motor function in both naïve and ERT-switch patients, as well as stability or improvements in forced vital capacity. The consistency and magnitude of improvements exceeded our expectations and follow the initial improvements seen on key biomarkers of muscle damage and disease substrate," said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. "These preliminary functional results are very encouraging and suggest a clinically meaningful improvement for patients.  We look forward to additional data from all patients in the third quarter as we continue in our mission to develop an improved treatment option for people living with Pompe disease."...

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The IPA Board was extremely saddened to learn of the unexpected passing of Henri Termeer, former CEO of Genzyme. Every tribute to Henri has mentioned the patient-centered focus he instilled and fostered within Genzyme. From personal experience, the IPA fully agrees with this characterization. The Pompe community has been extremely fortunate to have had Genzyme and Henri Termeer as partners in developing a treatment for Pompe. In the Pompe Community we have a true partnership between the three stakeholders: the Medical/Scientific Community, Industry, and the Patient Community. This partnership is only possible when all parties respect and value each other. The IPA believes that Henri Termeer was instrumental in setting the tone for Industry partners to engage with, and learn from their counter-parts in the Patient Community. He will be greatly missed.

Read more

• Henri Termeer, key biotech leader who built Genzyme into an industry giant, dead at 71 (The Boston Globe)
• Bio Leaders Mourn Passing of Genzyme Exec, Orphan Drug Innovator Termeer (xconomy.com)
• Henri Termeer Obituary: Link 1, Link 1

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The IPA would like to thank everyone who participated in the Talent of Pompe (ToP) Awards Contest on the occasion of the Fourth Annual International Pompe Day!

The IPA is very excited to announce the winners in each category:

Age 0-5

First Place: Abdurrahman Amerat

Many people find it hard to breathe and if we save our trees we can then make it easier for people to breathe everywhere in the world!