It is difficult to say what is impossible,
for the dreams of yesterday
are the hopes of today
and the reality
of tomorrow.

Robert H. Goddard

The Acid Maltase Deficiency Association, A M D A, was formed to assist in funding
research and to promote public
awareness of Acid Maltase Deficiency,
also known as Pompe's Disease.

2006 AMDA/IPA Patient Conference Report

Acid Maltase Deficiency-- A Brief Summary 

AMDA's Advisory Board

AMDA's Review of Geeta Anand's "The Cure"

2004-2005 Initiatives for Pompe's Disease

2005 AGSD-UK Conference Report

Listing of All Clinical Studies for Pompe's Disease--(Type in "Pompe")

• Information on Late-onset screening study now available

IPA Patient Questionnaire (Still in progress)

• Article on the Results of the IPA/Erasmus Patient Questionnaire

• Second Article on the Results of the IPA/Erasmus Patient Questionnaire

• Third Article on the Results of the IPA/Erasmus Patient Questionnaire

Physician's Guide to Pompe's Disease--Free Pamphlet from NORD

AMDA's Pompe's Disease Tele-Communication Program (new Recording added)

AMDA's Pompe Mentoring Program

Respiratory Support Information

• Flying with a Ventilator in the United States

Pompe Center of the Erasmus Medical Center Rotterdam, The Netherlands-- (Contains mutation database)

Meet the Patients--(Story of a Patient in the Clinical Trials) 

AMDA Patient Registry

Research Publications on Pompe's Disease

Research Developments

• Scientific Board Formed
• Research Highlights
• AMDA Projects
• AMDA Patient Conferences -- 2001 and 2006

Clinical Trials

• General Information
  
• January 1999--Sophia Children's Hospital/Erasmus University, The Netherlands
  Clinical Trials Begin--Pharming Group N.V.

• June 1999--Duke University Medical Center-U.S.A.
  Clinical Trials Begin--Synpac Pharmaceuticals Limited

• March 2001--Genzyme/Pharming/Synpac--Joint Venture Formed--Technology Purchased
   2000-2001-Clinical Trials--Exclusive Interview with Genzyme

• May 2001--Novazyme Pharmaceutical, Inc., U.S.A.
   Clinical Trials Planned--Exclusive Interview with Novazyme

• August 2001--Article on Transgenic Enzyme Replacement Therapy

• August 2001--Genzyme/Novazyme Merger

• October 2001-- Genzyme Acquires Pharming Manufacturing Facility

• April 2002-- Genzyme Press Release

• July 2002--Genzyme Begins Phase Out of Enzyme Produced in Milk of Transgenic Rabbits

• March 2003-- AMDA's Response to Article in MDA's "Quest"

• March 2003-- Genzyme Enrolling Patients in New Clinical Trial (Study 1702)

• May 2003-- Genzyme Enrolling Patients in New Clinical Trial (Study 1602)

• January 2004 --AMDA Newsletter Detailing Genzyme Initiatives in 2004

• April 2004 -- Suspension of Late-Onset Expanded Access Program

• August 2004 -- Joint IPA/Genzyme Statement announcing the new status of the Expanded Access Programs

• December 2004 -- December 2004 Update of Genzyme's Pompe Project

• December 2004 -- Genzyme Files for European Approval of Myozyme® for Treatment of Pompe Disease

• March 2005 -- March 2005 Update of Genzyme's Pompe Project

• April 2005 -- Genzyme Reports Interim Results from Pivotal Study of Myozyme®

• July 2005 -- July 2005 Update of Genzyme's Pompe Project

• July 2005 -- Genzyme Files for FDA Approval of Myozyme® for Treatment of Pompe Disease

• September 2005 -- September 2005 Update of Genzyme's Pompe Program

• January 2006 -- Genzyme Provides Update on U.S. Marketing Application for Myozyme®

• April 2006 -- European Commission Approves Genzyme's Myozyme®

• April 2006 -- FDA Approves Genzyme's Myozyme®

• June 2006 -- Myozyme's® Label available for patients to review

• January 2007 -- January 2007 Update from Genzyme on the LOTS trial

• April 2007 -- Genzyme Pompe Program Update

• May 2007 -- Details Concerning the Myozyme Temporary Access Program (MTAP)

• May 2007 -- Pompe Program Update from Genzyme

• June 2007 -- June 2007 Pompe Program Update from Genzyme

• June 2007 -- Updated information on the Myozyme Temporary Access Program (MTAP)

• October 2007-- October Update from Genzyme on Myozyme Supple

• January 2008 -- January Pompe Program Update from Genzyme--Important Information Inside

• April 2008 -- Important Update on Myozyme Supply Situation-- April 21, 2008  

• April 2008 -- April 2008 Pompe Program Update--April 22, 2008

• June 2008 -- Amicus Therapeutics Begins Phase 2 Clinical Trial of AT2220 in Pompe Disease

• November 2008 -- November 2008 Pompe Program Update
  


• November 2008 -- Myozyme Produced at the 2000 L Bioreactor Scale to Receive Accelerated Approval

• November 2008 -- November 2008 Pompe Program Update--November 19, 2008

• January 2009 -- Myozyme Supply Update January 2009 from Genzyme--Global version
  

• January 2009 -- Myozyme Supply Update January 2009 from Genzyme--United States version

• January 2009 -- EMEA Press Release concerning Myozyme supply situation--January 16, 2009

• January 2009 -- Guidance to the Pompe Community on the Management of Myozyme Supply
  develpoed by the Myozyme Stakeholders Working Group

• January 2009 --  IPA Statement Regarding the Management of Myozyme Supply--advice to Patient Groups

• January 2009 -- IPA Statement Regarding the Management of Myozyme Supply--advice to Pompe Patients

• January 2009 -- AMDA Statement Regarding the Management of "Myozyme" supply

• January 2009 -- Frequently Asked Questions complied by the IPA
  

• February 2009 -- Genzyme Receives Positive Opinion on Myozyme (2000L) from CHMP in Europe (new)

• February 2009 -- European Authorities Approve Larger-Scale Production of Genzyme's Myozyme  (new)

• March 2009 -- Pompe Program Update from Genzyme (new)

• March 2009 -- Genzyme Receives Complete Response Letter from FDA on Lumizyme Application (new)

• March 2009 -- IPA Statement: The End of Myozyme Supply Restrictions (new)

IPA--International Pompe Association

• IPA News

• First IPA Conference--July 1999

• IPA Board Meeting--February 2002

• Letter from IPA Chairman--February 2002

• Letter from Vice President of Genzyme--March 9, 2002

• IPA/Genzyme Meeting--April 16-17, 2002

• IPA/Genzyme News--September 24, 2002

• IPA Patient Questionnaire--November 2002 (Still in Progress) 

• IPA/Genzyme News--December 2002

• IPA Conference --October 30-November 1, 2003

• Summary of 2004 International Pompe Association Conference

• The IPA has developed a Pompe Connections Brochure--October 2005

• Summary of the 2005 AFM-IPA Conference available

• Letter from IPA and Genzyme concerning LOTS

Memorials

Other Webs of Interest

AMDA Guest Book