Maze Therapeutics Starting Phase I Clinical Trial with MZE001, A Substrate Reduction Therapy
Recently, preclinical data were presented at the 18th Annual WORLDSymposium in San Diego, CA, supporting MZE001 as a treatment for Pompe disease.
John F. Crowley
John Crowley, Chairman of the Board and CEO of Amicus Therapeutics, discussing family, challenges when entering adulthood, newborn screening and what’s new at Amicus.
Naresh Kumar Meena and Nina Raben
Leading researchers from the NIH discuss the discovery of Pompe disease, Pompe disease research studies at the NIH, and the recognition of a new phenotype for Pompe disease following the introduction of enzyme replacement therapy.
Amicus Therapeutics establishes a 75,000 sq. ft. Gene Therapy Center of Excellence
Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.