We’re proud of our legacy in lysosomal storage disorders: a group of rare, genetic conditions caused by enzyme deficiencies. Working closely with patient communities, we’ve been developing pioneering medicines for Fabry, Gaucher, and Pompe diseases, and advancing toward new treatments for patients with acid sphingomyelinase deficiency (ASMD) and GM2 gangliosidoses (Tay-Sachs disease, AB variant, and Sandhoff disease).
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Client Success Team (CRM),
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