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New registry aims to unlock secrets of rare childhood diseases

NCT ID NCT05619900

Recruiting now Knowledge-focused Sponsor: University of California, San Francisco Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study collects information from up to 250 patients with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to understand how these conditions develop and respond to treatments given before birth. Researchers will track symptoms, lab results, and organ function over time. The goal is to improve prenatal care and outcomes for affected children.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

Gaucher disease Gaucher disease type II glycogen storage disease due to acid maltase deficiency, infantile onset inborn errors of metabolism lysosomal storage disease mucopolysaccharidosis Mucopolysaccharidosis I Mucopolysaccharidosis IV mucopolysaccharidosis type 1 mucopolysaccharidosis type 2 mucopolysaccharidosis type 4A mucopolysaccharidosis type 6 mucopolysaccharidosis type 7 Wolman disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MUCOPOLYSACCHARIDOSIS I MUCOPOLYSACCHARIDOSIS II MUCOPOLYSACCHARIDOSIS IV A MUCOPOLYSACCHARIDOSIS VI MUCOPOLYSACCHARIDOSIS VII NEURONOPATHIC GAUCHER DISEASE POMPE DISEASE INFANTILE-ONSET WOLMAN DISEASE

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • University of California San Francisco

    RECRUITING

    San Francisco, California, 94143, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact