2021 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 2021 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Dwayne’s Story
December 2021 Hi everyone, my name is Dwayne; I am 53 years old. I was diagnosed with Late Onset Pompe disease (LOPD) in November 2018 when I was 50 years old. I live in Southern California with my wife and mother-in-law. I am a father to four sons. I also have a...
Haley’s Story
November 2021 My name is Haley Hayes. I am from Virginia. I am 16 years old and was diagnosed with infantile-onset Pompe DiseaseA severe form of Pompe disease that begins in infancy and often affects the heart and muscles. at 6 months old. I will be sharing my...AMDA and Spark Webinar: Understanding Gene Therapy Research and the Potential Applications for Late Onset Pompe Disease
To watch the Webinar, please click here!
Caitlin’s Story
August 2021 My name is Caitlin Naldoza. I am 17 years old and was diagnosed with late onset Pompe Disease at 5 years old. I will be sharing my experience being a teenager living with Pompe and how it has affected me over the years. In middle school, that was when I...Re-Scheduled–Friday, Oct 8, 2021: AMDA and Spark Webinar: Understanding Gene Therapy Research and the Potential Applications for Late Onset Pompe Disease
Hi All, We are excited to announce that the webinar is on for Friday, October, 8, 2021 at 1 PM CST. If you have already registered, you don’t need to do anything. If you have NOT registered yet, it’s not too late! Please click here to register. And,...FDA approves Sanofi’s Nexviazyme® (avalglucosidase alfa-ngpt) for Late-Onset Pompe
PARIS – August 6, 2021 – The U.S. Food and Drug Administration (FDA) has approved Nexviazyme® (avalglucosidase alfa-ngpt) for the treatment of patients one year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that...
JOHN’S STORY
A year ago today [April 29, 2004] our son, John, passed away from Pompe Disease. He was four years old. Most of you will not recognize his name, but you all should know who he is. John was patient 101 – the first person to receive the CHO Enzyme Replacement Therapy...