Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trialA research study that tests new treatments or approaches in people. More for gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More for Pompe. To read the Pompe Program Update, please click here.

Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trialA research study that tests new treatments or approaches in people. More. Please click here to read.

As everyone knows, due to the ongoing COVID-19 pandemic, the Patient-Focused Drug Development Meeting (PFDD) that was originally scheduled for March 09, 2020, was postponed. It has been moved to a Virtual Meeting that will take place Monday, July 13, 2020, from...

Update: July 8, 2020 It is with great sadness and disappointment that we hereby announce the cancellation of the 2020 Pull For Pompe Shoot due to the on-going COVID-19 pandemic.When we initially postponed the Shoot to August 15, we had high hopes that the virus would...

With all of the questions and uncertainty surrounding the ongoing COVID-19 pandemic, the AMDA worked with Dr. Priya Kishnani and her group at Duke University on the following Question and Answer Fact Sheet. We hope that the Pompe Community will find the information...

Dear IPA Board of Directors, As I’m sure you are aware, the World Health Organization (WHO) has declared a pandemic in response to the COVID-19 (SARS-CoV-2) outbreak. Given this serious health situation, the US Centers for Disease Control and Prevention (CDC)...

Please click here to read a statement from Amicus Therapeutics regarding COVID-19.

Announcement Regarding COVID-19 and the Pull for Pompe (03/18/2020): After careful consideration, and long discussions among the organizers; the 2020 PCMA Pull for Pompe has been postponed until Saturday, August 15, 2020. The Team of volunteers for The Pull For Pompe...

AMDA WEBINAR Title:  The common c.-32-13T>G splicingA normal step in how the body reads gene instructions. If splicing goes wrong, the wrong protein may... More variant of GAA geneThe gene responsible for making the GAA enzyme. More: from functional characterization to the identification of new therapeutic targets for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. Date:  January 23, 2020 Time:  9:30am PT, 11:30am CT, 12:30pm ET Register Now: ...

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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More to share their perspective and inform...

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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...

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...

Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Pivotal Study of AT-GAA in Patients with Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Global Study to Assess AT-GAA Compared to Standard-of-Care Enzyme Replacement Therapy (ERT)A treatment that replaces the missing enzyme through IV infusion. More over 52 Weeks CRANBURY, N.J., Dec. 20, 2018— Amicus...

Spark Therapeutics Announces New Preclinical Data for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. More 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...

SPARK THERAPEUTICS GENE THERAPYA developing treatment that aims to fix, replace, or add genetic instructions. More RESEARCH WEBINAR Please join the AMDA for a webinar focused on Spark Therapeutics and gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More research Take this opportunity to learn more about Spark as a company and the field of gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More research. We will be joined by...