Patient Focused Drug Development Meeting to be held March 09, 2020
The AMDA is excited to announce that it is partnering with the MDA to host a Patient Focused Drug Development Meeting (PFDD) on March 09, 2020. The PFDD meeting is an opportunity for patients and families living with Pompe disease to share their perspective and inform...
2019 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2019 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
2019 AMDA/IPA International Conference
The AMDA/IPA International Pompe Patient and Scientific Conference will take place in San Antonio, Texas from October 25-27, 2019. Please click here for the 2019 Conference Registration Form. Each family, speaker, patient organization representative, industry...
2019 PCMA’s Pull For Pompe!
The AMDA is excited to announce that the 2019 PCMA’s Pull for Pompe will take place on Saturday, April 13th at the National Shooting Complex in San Antonio, Texas! This is the 9th Annual Pull for Pompe hosted by the PCMA. The Pull for Pompe clay shooting event is...
Press Release: Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Pivotal Study of AT-GAA in Patients with Pompe Disease Global Study to Assess AT-GAA Compared to Standard-of-Care Enzyme Replacement Therapy (ERT) over 52 Weeks CRANBURY, N.J., Dec. 20, 2018— Amicus...
Spark Therapeutics Gene Therapy Research Webinar
Spark Therapeutics Gene Therapy Research Webinar Download an audio only version
Press Release: Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy
Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate Data from IND-enabling studies in three species support moving SPK-3006 into the clinic in 2019 PHILADELPHIA, Oct. 08, 2018—Spark Therapeutics, a fully integrated, commercial...
AMDA Webinar: Spark Therapeutics Gene Therapy Research
SPARK THERAPEUTICS GENE THERAPY RESEARCH WEBINAR Please join the AMDA for a webinar focused on Spark Therapeutics and gene therapy research Take this opportunity to learn more about Spark as a company and the field of gene therapy research. We will be joined by...
2018 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2018 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Press Release: Amicus Announces Regulatory and Clinical Updates for AT-GAA in Pompe Disease
CRANBURY, N.J., Sept. 10, 2018—Amicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, announced today regulatory and clinical advancements in its development program AT-GAA for...
Press Release from Erasmus Medical Center: Making Muscle from Skin
Researchers from the Erasmus MC in Rotterdam, Netherlands, have succeeded in generating muscle cells from skin cells. This is significant as skin cells can be relatively easily obtained from a skin biopsy. The team developed a protocol to obtain pure muscle cells (ie...
2017 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2017 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease
CONCORD, Mass., July 11, 2017—Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic diseases, today announced that it has initiated dosing in a Phase 1/2 clinical trial evaluating VAL-1221 in...
In Memory of Henri Termeer
he AMDA was saddened to learn of the sudden passing of Henri Termeer, former CEO of Genzyme. We have known and worked with Henri since the late-90s through his role as the CEO of Genzyme. We will never forget how he always took the time to talk to patients and their...
Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Phase 1/2 Study
Excerpt . . . . CRANBURY, N.J., May 15, 2017 —Amicus Therapeutics 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...
Pompe in the News: What Happened to the Truth?
On February 28, 2017—Rare Disease Day—Pompe disease was once again the subject of national media attention. During his Joint Address to Congress, President Trump brought national attention to patients suffering from rare diseases in general, and Pompe disease in...
2017 PCMA of Texas Pull for Pompe!
The AMDA is excited to announce that the 2017 PCMA of Texas Pull for Pompe will take place on Saturday, March 25th at the National Shooting Complex in San Antonio, Texas! This is the 7th Annual Pull for Pompe hosted by the PCMA of Texas. The Pull for Pompe clay...
2016 Helen Walker Research Grant Awarded
The seventh annual AMDA Research Grant (the 2016 Helen Walker Research Grant) was awarded to Dr. Frédéric Lofaso with the Hôpital Raymond Poincaré in France for his Project: “Acid Maltase Deficiency Myopathy Follow-Up.” The aim of this project is to improve the...
Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy
Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease Cambridge, Mass. – November 4, 2016 - Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first patient has been...
2016 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2016 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...