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Advancing Integrated Therapies for Gaucher Disease

RecruitingN/A

Sponsor: Yale University

Conditions: Gaucher Disease

Countries: United States

The purpose of this study is to better understand the natural history, clinical outcomes, and biological features of Gaucher disease in patients receiving standard medical care

Eligibility overview

Sex: ALL

Age: 3 Months to 75 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria

Inclusion Criteria:

Aim 1

* Confirmed diagnosis of GD2 or GD3 based on genotype and phenotype
* Evidence of pulmonary infiltrative disease and/or mediastinal or mesenteric lymphadenopathy
* Receiving or eligible for standard-of-care therapy
* Age ≥ 3 months
* Ability to provide informed consent (or parental consent with assent as appropriate)

Aim2

* ages 10-75
* persistent skeletal disease despite long-term therapy

Aim3

* Gaucher disease and clinical features of Parkinson disease
* Ability to provide informed consent

Exclusion Criteria:

Aim 1

* Inability to comply with observational follow-up
* Any condition that, in the investigator's judgment, precludes safe participation

Locations (1)

New Haven, Connecticut, United States