Mucopolysaccharide Type I (MPS-I) belongs to the wider group of lysosomal storage disorders. The most serious form is Hurler’s syndrome. None of the available interventions for MPS-I modify central nervous system pathology, while the proposed drug reaches CNS. The subject is not yet an approved drug, but it is now being tested in several clinical trials for common diseases, such as stroke. Its low toxicity and favourable pharmacokinetic behaviour make it promising for a smooth and rapid transition for treatment of MPS-I patients, possibly in combination with the available therapeutic options.
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