- Nov 2, 2022

Intracerebroventricular dosing of N-sulfoglucosamine Sulfohydrolase Inmucopolysaccharidosis Mice

Article In the Press

Journal of Biological Chemistry: Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase inmucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction

Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations

Article In the Press The Journal of Pharmacology and Experimental Therapeutics: Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis

Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB

Article In the Press The Journal of Pediatrics: Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB

Marcus Rashford and Chris Martin join mother’s plea for son, 9, to access ‘life-saving’ trial drug

A mother is desperately seeking access to a trial drug for her nine-year-old son that she hopes could save his life. Severine Hamilton is trying to obtain a prototype enzyme replacement therapy for he

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Intracerebroventricular dosing of N-sulfoglucosamine Sulfohydrolase Inmucopolysaccharidosis Mice

Article In the Press

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