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Condition
Mucopolysaccharidosis Type 3
Symptoms, Doctors, Treatments, Research & More
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Study
Condition:
Sanfilippo Syndrome Type A
Journal:
Molecular genetics and metabolism
Treatment Used:
Intrathecal Heparan-N-Sulfatase (HNS)
Number of Patients:
21
Published —
December 12, 2018
This study reported on the effectiveness , pharmacokinetics, safety, and tolerability of intrathecal (IT) administration of recombinant human heparan-N-sulfatase (rhHNS) in the treatment of Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA), a lysosomal disorder associated with progressive neurodegeneration in early childhood.
