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634 results for “MPSI”

Trials

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Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

3 participants · First posted Nov 27, 2012

A Natural History Study of Sanfilippo Syndrome Type D

10 participants · First posted Dec 13, 2022

Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

First posted Dec 27, 2022

Drugs

View all 79 →

MPSP-001

Epsi-gam

CPSI-1306

Organizations

MSI ASIA PACIFIC

Australia

MSI Methylation Sciences Inc

Canada

News

View all 51 →

Blood-Brain Barrier Breakthrough: Novel Delivery System Shows Promise for Brain Disease Treatment

Ultragenyx to Pursue Accelerated Approval for UX111 Gene Therapy in MPSIIIA

RGX-121 Gene Therapy Shows Sustained Efficacy in Hunter Syndrome Trial

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