Aro Biotherapeutics to Present New Preclinical Data
Aro Biotherapeutics to Present New Preclinical Data Highlighting the Potential of Centyrin-siRNA Conjugates for the Treatment of Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. To read the...
AMDA Zoom Webinar With Sanofi
Webinar Details Title: POMPE REGISTRY: 18 YEARS OF LEADERSHIP AND CONTRIBUTIONS Date: Monday, April 11, 2022 Speaker:Joseph Bender, MD, MBA, Global Head of Rare Disease Registries Danielle Dong, ScM, CGC, Global Operations and Advocacy Lead Rare Disease Registries...2022 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 11th Annual PCMA’s Pull for Pompe will take place on Saturday, April 30, 2022 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...
McKenna’s Story
January 2022 I am McKenna Wellner and I was diagnosed with Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More at 19 years old and I am now 20. I first noticed symptoms the beginning of 2019 and...
Genevieve’s Story
January 2022 My name is Geneviève, and I am 42 years old. I was diagnosed with Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More at the age of 40. Here is my story. In my early thirties, I was...
Elizabeth’s Story
January 2022 I was diagnosed with LGMD when I was 12 years old. I had difficulty all my life with running, going upstairs, and doing sports, but no matter how hard things were I always pushed myself. I started working at 12 years old, started babysitting. At 16 years...
Margo’s Story
January 2022 This is Margot. She is almost two years old from Kentucky. She loves to swing, paint, and keep up with her 4-year-old brother. When she was born, there were no concerns. We went home and adjusted to our new life with baby number two. 8 days later, we...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 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...