TOP NEWS
Tiffany House Receives RareVoice Award for State Advocacy
We are honored to share this moment recognizing Tiffany House as a recipient of the RareVoice Award for State Advocacy. The award is presented by the EveryLife Foundation For Rare Diseases.This award is given to advocates whose voices have helped shape meaningful...
OTHER NEWS
Audentes Pompe Program Update
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. for gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. for Pompe. To read the Pompe Program Update, please click here.
Spark Therapeutics Clinical Trial FAQ
Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trial. Please click here to read.
Patient Focused Drug Development Meeting—UPDATE
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...
2020 PCMA Pull For Pompe!—Cancelled
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...
AMDA Q and A with Dr. Priya Kishnani on COVID-19 and Pompe
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...
Statement from Spark Therapeutics on COVID-19 and their Pompe Program
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)...
Statement from Amicus regarding COVID-19
Please click here to read a statement from Amicus Therapeutics regarding COVID-19.
10th Annual PCMA Pull for Pompe!
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...
Upcoming Webinar: January 23, 2020
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 be made. variant of GAA geneThe gene responsible for making the GAA enzyme.: 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 that damages muscles and can affect breathing and, in some cases, the heart.. Date: January 23, 2020 Time: 9:30am PT, 11:30am CT, 12:30pm ET Register Now: ...
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...
Acid Maltase Deficiency Association
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