Pompe Disease
Medicalert services for individuals with pompe.
Pompe disease. Pompe disease is an inherited lysosomal storage disorder lsd caused by a deficiency of the enzyme acid alpha glucosidase gaa. Reduced or absent levels of gaa lead. Male or female subjects 18 to 50 years old. Have a diagnosis of pompe disease as defined by protein assay andor dna sequence of the acid alpha.
Amicus therapeutics announces regulatory and clinical updates for at gaa in pompe disease cranbury nj sept. 10 2018 globe newswire amicus therapeutics. William canfield is a glycobiologist chief scientific officer and founder of an oklahoma city based biotechnology company novazyme which was acquired by genzyme in. 232300 glycogen storage disease ii.
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