SETTING: Three referral medical centers. PATIENTS: All patients with glycogen storage disease type III who were followed in 1990 and for whom both immunoblot analysis and clinical data were available.

Through its Genzyme unit, Sanofi is one of the leading providers of drug therapies for Pompe disease, a glycogen storage disorder caused by a deficiency in the enzyme acid alfa glucosidase (GAA ...

Miglustat is already approved as Galafold in Europe and the US to treat Fabry disease, which like Pompe, is a glycogen storage disease ... version of the enzyme alpha-glucosidase, which is ...

Glycogen storage disease (GSD) refers to a group of rare genetic conditions that affect how the body stores and breaks down glycogen. The most common type is type I, also called von Gierke disease.

Philadelphia-based Aro Biotherapeutics’sABX-1100 is designed to deliver a glycogen ... Pompe disease community has long awaited the development of a treatment that overcomes the limitations of ...

of the genetically deficient enzyme, ABX1100 has shown significant reductions in glycogen in preclinical models and may reduce the need for frequent infusions of ERT. “The Pompe disease ...

Pompe disease or Glycogen Storage Disease is a rare genetic disease caused by the buildup of a sugar called glycogen in the body’s cells. It is caused by the deficiency or absence of the enzyme ...

a key enzyme required for synthesizing glycogen. ABX1100 is in early-stage development for the treatment of Pompe disease (Clinicaltrials.gov #NCT06109948) and has received Orphan Drug ...

Aro Biotherapeutics Announces U.S. FDA Clearance of IND Application for ABX1100 for Potential Treatment of Late-Onset Pompe Disease ... in the enzyme that breaks down muscle glycogen, a stored ...

"Attainment of these milestones bolsters our confidence as we continue to enroll patients with late-onset Pompe disease ... deficiency in the enzyme that breaks down muscle glycogen, a stored ...