The Official Website of the Acid Maltase Deficiency Association

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

Archives

2014 October

United States Pompe Community Update from Genzyme

Posted on October 17, 2014 - Filed under

2014 August

FDA and Genzyme Announce Label Expansion of Lumizyme in United States

Posted on August 01, 2014 - Filed under

2014 July

2014 Helen Walker Research Grant Now Accepting Applications

Posted on July 12, 2014 - Filed under

2014 May

BioMarin Doses First Patient in Phase 3 INSPIRE Trial with BMN 701 for the Treatment of Pompe

Posted on May 27, 2014 - Filed under

AMDA to Celebrate First International Pompe Day!!

Posted on May 04, 2014 - Filed under

2014 February

2014 AMDA Pull for Pompe

Posted on February 23, 2014 - Filed under

Update on Genzyme’s neo-GAA Pompe Clinical Development Program

Posted on February 11, 2014 - Filed under

2014 January

2013 Helen Walker Research Grant Awarded

Posted on January 01, 2014 - Filed under

2013 July

2013 Helen Walker Research Grant Applications

Posted on July 22, 2013 - Filed under

Update on Genzyme’s neo-GAA Program

Posted on July 16, 2013 - Filed under

2013 PCMA of Texas’s Pull for Pompe a HUGE Success!

Posted on July 01, 2013 - Filed under

2013 May

Federal Advisory Committee Recommends Pompe Disease for Newborn Screening

Posted on May 17, 2013 - Filed under

Callidus Biopharma Announces $ 4 .6 million in Series A Financing to Pursue Orphan Disease Therapies

Posted on May 13, 2013 - Filed under

2013 March

BioMarin to Advance BMN-701 for Pompe Disease to Next Phase of Development

Posted on March 22, 2013 - Filed under

2013 February

2013 PCMA of Texas’s Pull for Pompe set for April 20, 2013!

Posted on February 16, 2013 - Filed under

2013 January

2012 AMDA Research Grant Awarded

Posted on January 05, 2013 - Filed under

Amicus Announces Positive Results from Phase 2 Chaperone-ERT Co-Administration Study

Posted on January 04, 2013 - Filed under

2012 November

Pompe Community Loses Valued Advocate

Posted on November 30, 2012 - Filed under

2012 October

2012 AMDA/Genzyme Patient Diagnosis Survey Results

Posted on October 29, 2012 - Filed under

2012 September

Dr. Arnold Reuser Decorated by Queen of Netherlands

Posted on September 04, 2012 - Filed under

2012 July

2012 AMDA Research Grant

Posted on July 30, 2012 - Filed under

2012 PCMA Pull for Pompe Fundraiser Results

Posted on July 25, 2012 - Filed under

2012 May

BioMarin Pompe Program Update

Posted on May 14, 2012 - Filed under

2012 April

2011 AMDA/IPA Conference DVDs available!

Posted on April 09, 2012 - Filed under

2012 March

March 2012 Community Update From Genzyme Regarding 160L Myozyme Supply

Posted on March 20, 2012 - Filed under

2012 February

2012 PCMA Pull for Pompe Fundraiser

Posted on February 26, 2012 - Filed under

2012 January

Announcement Regarding 160 liter Myozyme Supply

Posted on January 17, 2012 - Filed under

2011 December

Inspired By Pompe Cookbooks available for Sale!

Posted on December 06, 2011 - Filed under

Amicus Therapeutics Announces First Patient in Phase 2 Study for Pompe Disease

Posted on December 01, 2011 - Filed under

2011 August

Support for Proposed Legislation in California Regarding Prior Authorization

Posted on August 30, 2011 - Filed under

2011 July

2011 AMDA Pompe Disease Research Grant Opportunity

Posted on July 12, 2011 - Filed under

2011 May

Upcoming Event: Respiratory Muscle Strength Training in Pompe Disease Webinar

Posted on May 20, 2011 - Filed under

2011 March

PCMA of Texas hosting “Pull for Pompe” Fundraiser

Posted on March 18, 2011 - Filed under

Cincinnati Children’s Hospital Study on Speech, Language and Swallowing Problems

Posted on March 09, 2011 - Filed under

2011 January

AMDA Webinar on Genzyme’s Next Generation Pompe Treatment: Clinical Development Overview

Posted on January 31, 2011 - Filed under

BioMarin Initiates Phase 1/2 Trial for BMN 701 for the Treatment of Pompe Disease

Posted on January 21, 2011 - Filed under

Genzyme to Build Additional Plant to Support Growth of Myozyme® and Lumizyme®

Posted on January 21, 2011 - Filed under

2010 AMDA Research Grant Awarded to Dr. Andrea Amalfitano

Posted on January 01, 2011 - Filed under

2010 August

BioMarin Receives Orphan Drug Designation from FDA for Pompe Disease

Posted on August 30, 2010 - Filed under

August 2010 Pompe Program Update from Genzyme

Posted on August 10, 2010 - Filed under

2010 July

AMDA 2010 Research Grant Opportunity

Posted on July 31, 2010 - Filed under

2010 June

Pompe Patient Meeting Hosted by Columbia University

Posted on June 14, 2010 - Filed under

2010 May

Pompe Program Update—FDA Approves Lumizyme

Posted on May 27, 2010 - Filed under

The FDA Approves Lumizyme for Late-Onset Pompe Disease

Posted on May 26, 2010 - Filed under

2010 January

Pompe Disease: The Real Story

Posted on January 21, 2010 - Filed under

AMDA Press Release Regarding Extraordinary Measures

Posted on January 20, 2010 - Filed under

2009 December

ATAP Inclusion/Exclusion and List of Study Sites

Posted on December 07, 2009 - Filed under

December 2009 Pompe Program Update

Posted on December 04, 2009 - Filed under Pompe Program Update,

2009 November

Influenza Vaccinations and Pompe Disease

Posted on November 27, 2009 - Filed under

Genzyme invites the Gaucher, Fabry, Pompe and MPS I communities to a Town Hall Meeting

Posted on November 21, 2009 - Filed under

November 2009 Pompe Program Update from Genzyme

Posted on November 17, 2009 - Filed under Pompe Program Update,

November 2009 Pompe Program Update from Genzyme

Posted on November 17, 2009 - Filed under Pompe Program Update,

2009 October

Amicus Therapeutics Announces Plan to Initiate Phase 1 Study of AT2220 for Pompe Disease

Posted on October 01, 2009 - Filed under

2009 July

Genzyme’s Second Quarter Earnings Statement

Posted on July 23, 2009 - Filed under

2009 May

Genzyme Submits All Information Requested by FDA for Lumizyme

Posted on May 22, 2009 - Filed under

IPA Statement: The End of Myozyme Supply Restrictions

Posted on May 08, 2009 - Filed under

2009 March

Pompe Program Update from Genzyme

Posted on March 02, 2009 - Filed under Pompe Program Update,

Genzyme Receives Complete Response Letter from FDA   on Lumizyme Application

Posted on March 02, 2009 - Filed under

2009 February

Amicus Therapeutics Suspends Enrollment for Phase 2 Clinical Trial of AT2220 for Pompe

Posted on February 27, 2009 - Filed under

European Authorities Approve Larger-Scale Production of Genzyme’s Myozyme

Posted on February 26, 2009 - Filed under

Genzyme Receives Positive Opinion on Myozyme from CHMP

Posted on February 19, 2009 - Filed under

New video added to “Meet the Patients”

Posted on February 04, 2009 - Filed under

November 2008 Pompe Program Update

Posted on February 04, 2009 - Filed under

Myozyme Produced at the 2000 L Bioreactor Scale to Receive Accelerated Approval

Posted on February 04, 2009 - Filed under

November 2008 Pompe Program Update 2

Posted on February 04, 2009 - Filed under

2009 January

IPA FAQs About the Myozyme Supply Problem

Posted on January 18, 2009 - Filed under

AMDA Statement Regarding the Management of “Myozyme” supply

Posted on January 17, 2009 - Filed under

PRESS RELEASE Priority access for children during Myozyme supply shortage

Posted on January 16, 2009 - Filed under

IPA Statement Regarding the Management of Myozyme Supply - Adivce To Pompe Patients

Posted on January 14, 2009 - Filed under

IPA Statement Regarding the Management of Myozyme Supply

Posted on January 14, 2009 - Filed under

Guidance to the Pompe Community on the   Management of Myozyme Supply

Posted on January 13, 2009 - Filed under

Myozyme Supply Update January 2009

Posted on January 01, 2009 - Filed under

MYOZYME® (2000 L) Supply Update – US Version – January 2009

Posted on January 01, 2009 - Filed under

2008 November

Genzyme Pompe Program Update—November 2008

Posted on November 17, 2008 - Filed under Pompe Program Update,

Genzyme Pompe Program Update—November 10, 2008

Posted on November 10, 2008 - Filed under Pompe Program Update,

2008 June

Amicus Therapeutics Begins Phase 2 Clinical Trial of AT2220 in Pompe Disease

Posted on June 04, 2008 - Filed under

2008 April

Genzyme Pompe Program Update—April 2008

Posted on April 21, 2008 - Filed under Pompe Program Update,

2008 January

January 2008 US Myozyme Supply Update

Posted on January 15, 2008 - Filed under

2007 December

Genzyme Study of Myozyme® for Late-Onset Pompe Patients

Posted on December 13, 2007 - Filed under

2007 October

Genzyme Pompe Program Update—October 2007

Posted on October 03, 2007 - Filed under Pompe Program Update,

2007 June

Genzyme Pompe Program Update—June 2007

Posted on June 27, 2007 - Filed under Pompe Program Update,

MTAP Fact Sheet June 2007

Posted on June 27, 2007 - Filed under

2007 May

MTAP Fact Sheet May 2007

Posted on May 02, 2007 - Filed under

2007 April

Genzyme Pompe Program Update—April 2007

Posted on April 24, 2007 - Filed under Pompe Program Update,

2006 June

Letter From IPA and Genzyme Concerning LOTS

Posted on June 07, 2006 - Filed under IPA News, Pompe Program Update,

2006 April

FDA Approves Genzyme’s Myozyme®  for All Patients with Pompe Disease

Posted on April 28, 2006 - Filed under Clinical Trials,

European Commission Approves Genzyme’s Myozyme®

Posted on April 03, 2006 - Filed under Clinical Trials,

2006 January

Genzyme Provides Update on U.S. Marketing Application for Myozyme

Posted on January 17, 2006 - Filed under Pompe Program Update,

2005 October

The IPA Pompe Connections Brochure

Posted on October 01, 2005 - Filed under IPA News,

2005 July

Genzyme Submits BLA for Myozyme®  for Treatment of Pompe Disease

Posted on July 29, 2005 - Filed under Clinical Trials,

2005 May

Summary of the 2005 AFM-IPA Conference

Posted on May 12, 2005 - Filed under IPA News,

2005 April

Genzyme Reports Interim Results from Pivotal Study of Myozyme

Posted on April 26, 2005 - Filed under Clinical Trials,

2004 December

Genzyme Files for European Approval of Myozyme® For Treatment of Pompe Disease

Posted on December 20, 2004 - Filed under Clinical Trials,

2004 October

Summary of 2004 International Pompe Association Conference

Posted on October 11, 2004 - Filed under IPA News,

2003 November

IPA Conference - October 30 - November 1, 2003

Posted on November 02, 2003 - Filed under IPA News,

2003 May

Genzyme Begins Enrolling Patients in Pivotal Pompe Disease Study

Posted on May 29, 2003 - Filed under Clinical Trials,

2003 March

Genzyme General Begins Enrolling Patients in Pompe Disease Study

Posted on March 19, 2003 - Filed under Clinical Trials,

2002 December

Genzyme’s Pompe Program Update

Posted on December 01, 2002 - Filed under IPA News, Pompe Program Update,

2002 October

IPA/Genzyme News—September 24, 2002

Posted on October 02, 2002 - Filed under IPA News,

2002 July

Transgenic Enzyme Production to Cease

Posted on July 04, 2002 - Filed under

2002 April

IPA/Genzyme Meeting

Posted on April 18, 2002 - Filed under IPA News,

Genzyme Press Release

Posted on April 17, 2002 - Filed under

2002 March

Letter from Vice President of Genzyme

Posted on March 05, 2002 - Filed under IPA News,

2002 February

IPA Board Meeting - February 18-19, 2002

Posted on February 20, 2002 - Filed under IPA News,

Letter from IPA Chairman to Pompe Patients

Posted on February 18, 2002 - Filed under IPA News,

2001 October

Genzyme Acquires Manufacturing Facility in Europe

Posted on October 04, 2001 - Filed under

2001 August

Genzyme to Acquire Novazyme Pharmaceuticals

Posted on August 07, 2001 - Filed under

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk

Posted on August 04, 2001 - Filed under

2001 May

Exclusive Interview with Novazyme

Posted on May 01, 2001 - Filed under Clinical Trials,

2001 March

Clinical Trials Genzyme/Pharming

Posted on March 31, 2001 - Filed under

Pharming and Genzyme to Start Study for Pompe’s Disease

Posted on March 30, 2001 - Filed under

Genzyme General and Pharming Report Publication of Study Results for Pompe Disease Therapy

Posted on March 21, 2001 - Filed under

Genzyme/Pharming/Synpac—Joint Venture Formed—Technology Purchased

Posted on March 01, 2001 - Filed under Clinical Trials,

2000 October

Genzyme General and Pharming Group Report Results From First Two Clinical Trials for Pompe Disease

Posted on October 06, 2000 - Filed under

Excerpt from presentation of Y.T. Chen ,M.D. Ph.D. (Duke University) at ASHG meeting

Posted on October 04, 2000 - Filed under

Statement from Genzyme and Pharming

Posted on October 01, 2000 - Filed under

2000 July

Results of First 36 Weeks of Treatment in Infants in Netherlands Published

Posted on July 30, 2000 - Filed under

2000 April

Genzyme General Obtains Rights to Pompe Disease Therapy from Synpac

Posted on April 20, 2000 - Filed under

2000 March

Pharming’s first Phase II clinical trial for Pompe’s disease finalised

Posted on March 15, 2000 - Filed under

1999 December

Enzyme Replacement Therapy (ERT), Gene Therapy Diet, Ephedrine, and Exercise Study

Posted on December 05, 1999 - Filed under Research Highlights,

1999 August

Systemic correction of the muscle disorder glycogen storage disease type II

Posted on August 04, 1999 - Filed under Research Highlights,

Duke Scientists Reverse a Rare Form of Muscular Dystrophy in Mice Using Gene Therapy

Posted on August 03, 1999 - Filed under Research Highlights,

1999 July

First IPA Conference Held July 2-4, 1999

Posted on July 04, 1999 - Filed under IPA News,

1999 June

Duke University Starts Clinical Trials for Pompe’s Disease

Posted on June 30, 1999 - Filed under Clinical Trials,

Modified Adenovirus for GSD-II

Posted on June 26, 1999 - Filed under Research Highlights,

1999 January

Phase II Pilot Trials Begin (January 1999) in the Netherlands

Posted on January 04, 1999 - Filed under Clinical Trials,

General Information On Clinical Trials

Posted on January 03, 1999 - Filed under Clinical Trials,

1998 October

Pharming and Genzyme Form Joint Venture to Develop Treatment for Pompe’s Disease

Posted on October 15, 1998 - Filed under

1998 July

Human Gene Therapy 9:1609-1616

Posted on July 22, 1998 - Filed under Research Highlights,

1998 June

Gene Therapy: National Institutes of Health

Posted on June 01, 1998 - Filed under Research Highlights,

1998 January

Netherland’s Scientists Produce Knockout Mouse Model of Pompe disease

Posted on January 31, 1998 - Filed under Research Highlights,

1996 November

Gene Therapy: Johns Hopkins University

Posted on November 30, 1996 - Filed under Research Highlights,

1996 October

Scientists Make Progress In Gene Therapy For Heart Disease

Posted on October 30, 1996 - Filed under Research Highlights,

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Address: AMDA PO Box 700248
San Antonio, Texas 78270 USA