Astellas Gene Therapies Press Release – Clinical Trial Update
“On June 26, 2022, Astellas Pharma, Inc. (“Astellas”) issued a press release announcing that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS Phase 1/2 clinical trialA research study that tests new treatments or...Voice of the Patient Report for the Pompe PFDD is Released!
The AMDA is excited to report that the final Voice of the Patient Report for the Pompe Patient-Focused Drug Development meeting has been released. To view the report, please CLICK HERE!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...