Recently, preclinical data were presented at the 18th Annual WORLDSymposium in San Diego, CA, supporting MZE001 as a treatment for Pompe disease.
An excellent review article focused on the latest developments in Pompe disease research was recently published by Naresh K. Meena and Nina Raben from the NIH.
Results from the COMET study were recently reported showing neoGAA (avalglucosidase alfa), the newer enzyme replacement therapy (ERT) currently in development, to be as effective as the currently approved ERT, Myozyme (alglucosidase alfa), in persons with late-onset Pompe disease.
Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.
Genzyme is in the early stages of investigating a next generation enzyme replacement therapy (neoGAA) for Pompe disease. It differs from the original ERT, whereby carbohydrates are bonded onto the enzyme during the complex manufacturing process of neoGAA. These carbohydrates are thought to help the enzyme enter the muscles more efficiently. Though it’s still very early in the development process and there are many unknowns, the hope is that through this more efficient uptake into the muscle cell, neoGAA might improve on the effectiveness of the existing therapy.
An article published on PubMed.gov, Dec. 2011, describes a 40-year old woman with Pompe Disease who successfully carried a healthy baby to term.