Walking distance improved in late-onset Pompe disease patients on enzyme replacement therapy (ERT),…
Articles by Aisha I Abdullah PhD
Fat replacement of back, abdominal, and trunk muscles in late-onset Pompe disease…
Administration of 3,4-diaminopyridine phosphate (3,4-DAPP) eased problems at the neuromuscular junction (NMJ) — sites of nerve-muscle communication…
The first patient has been dosed in the FORTISÂ Phase 1/2 trial of AT845, a single-dose gene therapy…
Early treatment with enzyme replacement therapy (ERT) and immune tolerance induction (ITI) greatly improved clinical outcomes for infants with…
A CRISPR gene-editing approach was used to correct two distinct Pompe disease mutations and to develop…
AVR-RD-03, Avrobio‘s investigational gene therapy for classic infantile-onset Pompe disease, was found to reduce…
Children with classic infantile Pompe disease treated for years with Myozyme (alglucosidase alfa, marketed as Lumizyme…
Fertility in women is not affected by late-onset Pompe disease (LOPD), but pregnancy may initiate or worsen…