First Patients Enrolled in Phase 2 Trial of SRT for Late-Onset Pompe Disease
Shionogi has announced the enrollment of the first patients in a global phase 2 clinical trial evaluating S-606001, an investigational oral therapy for adults with late-onset Pompe disease (LOPD). S-606001 is a substrate reduction therapy (SRT) designed to reduce...
Aro Biotherapeutics reports positive phase 1b results for ABX1100
Aro Biotherapeutics has reported positive topline results from its phase 1b clinical trial of ABX1100, an investigational therapy for adults with late-onset Pompe disease (LOPD). ABX1100 is a substrate reduction therapy (SRT) designed to reduce glycogen production in...
Aro Biotherapeutics starts Phase 1b Clinical Trial for Substrate Reduction Therapy ABX1100
Aro Biotherapeutics has launched a Phase 1b clinical trial for ABX1100, a substrate reduction therapy (SRT).
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.
New Review Article Highlights Latest Developments in Pompe Disease Research
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.
New ERT For Late-onset Pompe Disease Shows Promise
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.
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.
Update from Genzyme on neoGAA Pompe clinical development program 11th February 2014
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.
First experience with enzyme replacement therapy during pregnancy and lactation in Pompe disease
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.