Readeli®
INN: galsulfase
Description
Indications for use
Instruction
Galsulfase is an enzyme replacement therapy drug and represents a recombinant form of the natural human enzyme N-acetylgalactosamine-4-sulfatase. Patients with mucopolysaccharidosis type VI have a deficiency or absence of N-acetylgalactosamine-4-sulfatase. This enzyme normally catalyzes the hydrolytic cleavage of sulfate radicals from the glycosaminoglycan dermatan sulfate molecule.
Galsulfase is an enzyme replacement therapy drug that, when introduced into the body, restores the level of lysosomal enzyme activity sufficient to hydrolyze accumulated dermatan sulfate and prevent its further accumulation.
The drug Readeli® is indicated for long-term enzyme replacement therapy in adults and children with a confirmed diagnosis of Mucopolysaccharidosis Type VI (MPS VI, Maroteaux-Lamy syndrome).