At Sanofi’s inaugural Global Rare Impact Awards ceremony held on November 14, 2025, in Vienna, three extraordinary leaders were recognized

We don’t have a webinar scheduled at the moment, but more exciting sessions are on the way! Check back soon

Webinar Details Title: Overview of Breathing Muscle Weakness in Neuromuscular Disease (NMD) Date: Thursday, September 25, 2025 Speaker:Andrea Klein Webinar

The winner of the 2024 AMDA Helen Walker Research Grant: Dr. Jaymin Upadhyay, Boston Children’s Hospital, Harvard Medical School Project

Great news for the rare disease community—Texas has taken a big step by adding Pompe disease to its newborn screening

Webinar Details Title: The Role of Genetic Counselors on the Pompe Disease Care Team Date: Monday, June 9, 2025 Speaker:Erin

Tiffany Laurel House January 22, 1983 – May 25, 2025 Tiffany L. House, 42, of San Antonio, Texas, passed away

Webinar Details Title: The Pompe Survey: Past Insights and Future Goals Date: Tuesday, March 25, 2025 Speakers:Lauren Dobischok, MScMichelle Kruijshaar,

Webinar Details Title: Evolving our understanding of Pompe Disease – data driven insights Date: Wednesday, February 26, 2025 Speaker:Ryan Colburn