clinical trials

MZE001, an experimental oral substrate reduction treatment for Pompe disease, was well-tolerated in both single and multiple doses in a study conducted in healthy volunteers, according to Maze Therapeutics, the therapy’s developer. Based on the trial’s findings, Maze is planning to launch a new study in 2023 to…

A small study in the U.K. is evaluating a noninvasive MRI-based tool that could potentially detect late-onset Pompe disease (LOPD) without the need for invasive muscle biopsies. This specialized muscle MRI also would monitor glycogen buildup — a hallmark of the condition — in muscles over time. The…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD). The decision was due to a study participant developing peripheral sensory neuropathy — nerve damage outside the brain…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Treatment with the two-part investigational therapy AT-GAA improved walking ability and lung function for up to three years among adults with Pompe disease in a Phase 1/2 clinical trial. Barry Byrne, MD, PhD, from the University of Florida, presented the findings at the 2022 MDA Clinical & Scientific Conference,…

The investigational gene therapy ACT-101 overall was well-tolerated among three people with late-onset Pompe disease (LOPD) who were treated in a Phase 1/2 clinical trial. Moreover, the trial results also showed that treatment markedly increased levels of the GAA enzyme — deficient in Pompe disease — in the patients’ muscle…

The first healthy volunteers have been dosed in a Phase 1 clinical trial evaluating MZE001, Maze Therapeutics’ experimental oral therapy for Pompe disease. “The initiation of this study is a significant milestone for both Maze and the Pompe community as we advance into the clinic with a potentially…

Acceptable safety is being seen to date in four adults with late-onset Pompe disease (LOPD) given the one-time gene therapy  AT845 in the FORTIS Phase 1/2 trial, its developer, Astellas Gene Therapies, announced. Interim study data in these four enrolled patients, as of the Dec. 3 cutoff date, shows…

Maze Therapeutics has announced its plans to begin clinical testing of MZE001, its lead candidate therapy for Pompe disease, in the first half of this year. The California-based precision medicine company raised $190 million in financing — led by Matrix Capital Management — to support the development of…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…