First Participant Dosed in Phase 1/2 Gene Therapy Trial for Late-Onset Pompe Disease
AskBio has announced that the first participant has been dosed in a phase 1/2 clinical trial evaluating AB-1009, an investigational gene therapy for adults with late-onset Pompe disease (LOPD). AB-1009 is designed to address the underlying cause of Pompe disease by...
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...
Shionogi & Co., Ltd. and Maze Therapeutics, Inc. Announce Exclusive Worldwide License Agreement for MZE001
This Substrate Reduction Therapy drug could be the first pill taken by mouth to treat Pompe disease.
AVROBIO to Explore Strategic Alternatives
One of a handful of gene therapy companies with pipelines that include Pompe disease. Today AVROBIO announced a halt to their programs.
Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European Union
Amicus Therapeutics, announces the approval of Opfolda (miglustat) capsules, an enzyme stabilizer of cipaglucosidase alfa, by the European Commission for the treatment of Pompe disease.
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.
Avalglucosidase Alfa (Nexviazyme™) approved by Health Canada
Health Canada has approved avalglucosidase alfa (Nexviazyme™) for the long-term treatment of late-onset Pompe disease in patients over the age of 6 months.
FDA approves Nexviazyme® (avalglucosidase alfa-ngpt)
In a recent announcement, the Pompe disease community learned that the U.S. Food and Drug Administration (FDA) has approved Nexviazyme® for the treatment of late-onset Pompe patients that are one year old and older.