Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study

posted in: Uncategorized | 0

Amicus Therapeutics announced additional positive results from a global Phase 1/2 clinical study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen (disease substrate) in cells. Patients treated with ATB200/AT2221 for up to 12 months showed improvements in six-minute walk test (6MWT) distance and other measures of motor function, stability or increases in forced vital capacity (FVC), and durable reductions in biomarkers of muscle damage and disease substrate.

To Read the Complete Article at CheckOrphan, Click Here

Working on a cure for a rare disease? Need Breakthrough Therapy designation, Fast Track Designation, RMAT Designation, FDA or EMA Orphan Designations? The BioPharma Global Regulatory Team can help – We are the #1 US based not-for-profit Global regulatory affairs firm with cost-effective, time-efficient solutions to chaperone your path through the regulatory affairs process. Contact us at http://www.biopharmaglobal.com/contact/ or by phone +1(202)660-1826

Leave a Reply