Mucopolysaccharidosis Type VI clinical trials at University of California Health

3 in progress, 2 open to eligible people

Showing trials for

  • In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

    open to eligible females ages 18-50

    The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

    at UCSF

  • Registry of Patients Diagnosed With Lysosomal Storage Diseases

    open to eligible people ages up to 64 years

    This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.

    at UCSF

  • Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

    Sorry, in progress, not accepting new patients

    Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.

    at UCLA

Our lead scientists for Mucopolysaccharidosis Type VI research studies include .

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