Inês Martins, PhD, managing science editor —

Children receiving enzyme replacement therapy (ERT) for Pompe disease often experience immune reactions to treatment, presenting as rashes or severe allergy, sometimes after several successful infusions, a case series shows. While these hypersensitivity reactions can be avoided successfully with anti-allergy medications and longer infusion times in some patients, others…

Transplanting blood stem cells modified to carry the GAA gene raised glycogen levels almost to normal across a range of organs, including the brain, and improved locomotion in a mouse model of Pompe disease, a study reported. These findings suggest that blood stem cell gene therapy, in which a patient’s…