In an announcement at the International Annual Congress of the World Muscle Society, Amicus Therapeutics showed continuing positive results of their Phase 1/2 clinical study (ATB200-02) to investigate AT-GAA in both ERT-Naive and ERT-Switch patients with Pompe disease. Patients treated with AT-GAA for up to 18 months showed improvements in the six-minute walk test (6MWT) and other measures of motor function and muscle strength, stability or increases in forced vital capacity (FVC), and durable reductions in biomarkers of muscle damage. A very low rare of infusion associated reactions was also noted.
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- Rare Disease Day 2019
- Sarepta Therapeutics Enters into a Strategic Relationship with Aldevron in Support of Their Gene Therapy Program
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