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According to symptom onset age and disease severity, Pompe disease is classified into two forms: the infantile-onset form and the late-onset form (childhood-, juvenile-, and adult-onset forms).
L'article Daoudi anticipe une eventuelle baisse des prix a la pompe est apparu en premier sur ALBAYANE .
Data supporting the selection were presented at the 14th Annual WORLDSymposium 2018 in a poster titled: "A Novel Hybrid Promoter Directing AAV-mediated Expression of Acid Alpha-Glucosidase to Liver, Muscle, and CNS Yields Optimized Outcomes in a Mouse Model of Pompe Disease.
Pompe disease is caused by mutations in the gene that encodes the enzyme acid alpha-glucosidase, or GAA, which is responsible for degrading lysosomal glycogen.
Pompe Disease - Therapeutics under Development by Companies 12
Magglio was hardly alone: Most babies with Pompe disease who received the only available treatment soon produced antibodies that rendered it useless.
Crowley has had Pompe disease since she was 15 months old.
Elevated plasma cardiac troponin T levels caused by skeletal muscle damage in Pompe disease.
Pompe disease is a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA) affecting an estimated 50,000 people worldwide.
Mira el pompe, este es un juguete que Jack mismo invento ?
The report provides comprehensive information on the therapeutics under development for Pompe Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
DISCUSSION: Pompe disease, also known as glycogen storage disease type II, is an inherited disorder whose primary symptom is progressive weakness in the muscles used for mobility and breathing.