Treatment with the two-part investigational therapy AT-GAA improved walking ability and lung function for up to three years among…
Articles by Marisa Wexler, MS
The investigational gene therapy ACT-101 overall was well-tolerated among three people with late-onset Pompe disease (LOPD) who were treated in…
An investigational gene therapy called AT845 could effectively increase the activity of an enzyme that’s deficient in mouse and nonhuman…
Maze Therapeutics has announced its plans to begin clinical testing of MZE001, its lead candidate therapy for Pompe…
The European Medicines Agency has accepted applications from Amicus Therapeutics seeking approval of AT-GAA, a two-component investigational therapy for…
Nexviazyme (avalglucosidase alfa), a next-generation enzyme replacement therapy, has been approved by Health Canada for people with late-onset…
Training with continuous positive airway pressure therapy, known as CPAP, may help to normalize speech abnormalities in children with…
The U.S. Food and Drug Administration (FDA) is reviewing applications from Amicus Therapeutics asking for the approval of AT-GAA as…
Starting treatment with enzyme-replacement therapy (ERT) in the first days of life appears to lessen the risk of hearing…
The U.S. Food and Drug Administration (FDA) has approved Nexviazyme (avalglucosidase alfa), a next-generation enzyme replacement therapy for…