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wolman-disease Clinical Trials

A listing of wolman-disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 6 clinical trials
F Frederique Frisch

Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase Deficiency

Lipoprotein lipase (LPL) is an enzyme that plays an important role in removing triglycerides (TG) (molecules that transport dietary fat) from the blood. Patients with LPL deficiency (LPLD) display during their whole life very high plasma TG levels often associated with episodes of postprandial abdominal pain, malaise, blurred vision, dizziness …

18 - 75 years of age Both Phase N/A
T TUBA YILMAZ BULUT, Dr.

The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence

This study aimed to determine the effects of Kegel exercise and Ba Duan Jin applications applied to premenopausal women with urinary incontinence on quality of life and psychological well-being. Study Group of the Research: Premenopausal women between the ages of 45-55 who come to the family health center with any …

45 - 55 years of age Female Phase N/A
E Emma Canepa, MS, CCRP

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

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.

18 - 50 years of age Female Phase 1

Lysosomal Acid Lipase (LAL) Deficiency Registry

This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.

- 100 years of age Both Phase N/A
L Laurent Servais, MD, PhD

Baby Detect : Genomic Newborn Screening

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of …

- 28 years of age Both Phase N/A
E Emma Canepa, MS, CCRP

Registry of Patients Diagnosed With Lysosomal Storage Diseases

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.

- 64 years of age Both Phase N/A

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