Tralesinidase alfa for mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B)

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Year: 2020

Tralesinidase alfa is in clinical development for the treatment of patients with mucopolysaccharidosis type IIIB (MPS IIIB) also known as Sanfilippo syndrome type B. MPS IIIB is a rare and progressive genetic disorder caused by the deficiency of one of the enzymes needed to break down complex sugar molecules called mucopolysaccharides. Clinically, patients have behavioural problems, intellectual deterioration, sleep disorders, hearing impairment, facial dysmorphism, organomegaly, bowel disturbances, mild skeletal changes, and shortened life span. The clinical severity ranges from mild to severe.
Tralesinidase alfa is an investigational enzyme replacement therapy (ERT) designed to replace the faulty enzyme with a healthy one in patients with MPS IIIB. Tralesinidase alfa is delivered directly to the fluid surrounding the brain (cerebrospinal fluid). If licensed, tralesinidase alfa will offer new therapy option for patients with MPS IIIB who currently have no treatment options.