The Official Website of the Acid Maltase Deficiency Association

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Letter from IPA Chairman to Pompe Patients

Posted on: February 18, 2002

The International Pompe Association (IPA) board will meet on February 18-19th, 2002. Representatives of scientific/medical teams and Genzyme will attend. The following issues will be discussed:

1. Finalize the universal patient questionnaire to be distributed in the near future by the IPA to all member organizations throughout the world.

This questionnaire is a long-time endeavor by leading researchers in the Pompe field. It will serve a two-fold purpose:

A. To identify and classify Pompe patients worldwide
B. To serve as an independent patient database

This database will safeguard patient confidentiality, but will also allow accessibility to data by corporations, physicians, and medical facilities working towards treatments for Pompe’s disease.

2. Compassionate (or humanitarian) use will be discussed. The objective is to set up criteria for making enzyme replacement therapy available to patients before market approval is obtained.

Availability of enzyme is the critical issue in this agenda—how to make the currently produced enzymes available to more patients in the shortest possible time. This matter (expanded production) will be a major point of discussion with Genzyme representatives at this meeting.

3. Current results from the ongoing clinical trials (CHO and transgenic) and the current timetable for initiation of new CHO trials will be discussed.

4. Collaboration between IPA and Genzyme to release research and development information on a quarterly basis to patient organizations.

5. Expense of enzyme replacement therapy (when marketed) will be discussed—how to insure that the cost will not prohibit any patient from receiving treatment.

Be assured that patient organizations through the IPA are working with Genzyme as well as with Pompe researchers to continue to push for safe, effective, and timely treatment for all patients with Pompe’s disease.

Best Regards,
Randall House
President, IPA

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