Last Updated: 09/26/2026
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Found 20 publications
Urine N-Acetylaspartate Distinguishes Phenotypes in Canavan Disease.
Journal: Human gene therapy
Published: December 04, 2024
A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B.
Journal: The Journal of clinical investigation
Published: September 02, 2022
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: March 15, 2022
Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB.
Journal: The Journal of pediatrics
Published: January 05, 2022
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.
Journal: Drug delivery and translational research
Published: January 17, 2020
Best practices for the use of intracerebroventricular drug delivery devices.
Journal: Molecular genetics and metabolism
Published: January 13, 2018
Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.
Journal: Orphanet journal of rare diseases
Published: March 29, 2017
Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa.
Journal: JIMD reports
Published: February 17, 2017
Intracerebroventricular Delivery as a Safe, Long-Term Route of Drug Administration.
Journal: Pediatric neurology
Published: April 19, 2016
Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome.
Journal: Molecular genetics and metabolism
Published: March 29, 2016
Erratum to: Tolerability of High-Volume Subcutaneous Injections of a Viscous Placebo Buffer: A Randomized, Crossover Study in Healthy Subjects.
Journal: AAPS PharmSciTech
Published: April 24, 2015
Hypocalcaemia in patients with metastatic bone disease treated with denosumab.
Journal: European journal of cancer (Oxford, England : 1990)
Published: March 09, 2015
Last Updated: 09/26/2026