Registry of Patients with Lysosomal Storage Diseases

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Recruiting Observational Study
Mucopolysaccharidosis I Mucopolysaccharidosis II Mucopolysaccharidosis IV A Mucopolysaccharidosis VI Mucopolysaccharidosis VII Pompe Disease Infantile-Onset Neuronopathic Gaucher Disease Wolman Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
Up to 64
Sex
Any
Study type
Observational
Participants needed
250 (estimated)
Sponsor
University of California, San Francisco · Other
Who this trial is looking for

This trial is looking for patients diagnosed with lysosomal storage diseases and pregnant individuals whose fetus has been diagnosed with these diseases. Participants will help researchers understand the disease better and improve management for future patients.

Are You a Good Fit for This Trial?

You may be able to join if

  • I am aged 0-64 and diagnosed with a lysosomal storage disease.
  • I am pregnant and my fetus has been diagnosed with a lysosomal storage disease.

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

Think this trial could be right for you?

Answer three quick questions written from this study's own rules to see if you may qualify. No account needed to find out.

Check Your Eligibility
About This Trial

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.

Trial Locations
Loading…

Loading trial locations…

Eligibility Criteria
Inclusion Criteria: * Patients aged 0-64 with a diagnosis of a lysosomal storage disease * Pregnant patients whose fetus has a diagnosis of a lysosomal storage disease Exclusion Criteria: * There are no current exclusion criteria
Contacts

Billie Lianoglou, MS

415-476-2461

billie.lianoglou@ucsf.edu

CONTACT

Emma Canepa, MS, CCRP

415-476-7255

Emma.Canepa@ucsf.edu

CONTACT