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.

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Found 6 clinical trials
In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs). (IUERT)

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.

replacement therapy
  • 11 views
  • 17 Oct, 2022
  • 1 location
Problem-Solving for Rural Heart Failure Dyads

This study will develop and test the effectiveness of a culturally-sensitive, telephone-based, tailored dyadic problem-solving intervention to improve self-care in rural heart failure (HF) dyads. The target population is rural-residing HF dyads (patient and family caregiver). Rural dyads will be recruited from the Florida State University Institute for Successful Longevity …

Accepts healthy volunteers
heart failure
  • 0 views
  • 27 Jun, 2022
  • 8 locations
Clinical Trial of a Low Protein Diet in Patients With Cognitive Impairment

The wide-acting effects of Fasting-Mimicking Diets (FMDs) on metabolic, inflammatory and regenerative pathways leading to reduced pathology or risk factors for various diseases in mice and humans, has the potential to be effective against Alzheimer's disease (AD). It is proposed to conduct a randomized clinical trial of twelve monthly cycles …

  • 0 views
  • 04 Oct, 2022
  • 3 locations
Lysosomal Acid Lipase (LAL) Deficiency Registry

of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.

deficiency
lipase
  • 283 views
  • 04 Oct, 2022
  • 28 locations
Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)

responsible for hydrolyzing cholesterol esters and triglycerides that are delivered to lysosomes. Mutations in LIPA that completely inactivate LAL are the molecular cause of Wolman disease, a rapidly lethal

xanthelasma
low density lipoprotein
mixed hyperlipidemia
apolipoprotein
cholesterol esters
  • 3 views
  • 04 Oct, 2022
  • 4 locations
Abnormal Lipids - Causes and Effects

greater nerve damage compared with matched controls. LAL deficiency is underdiagnosed in patients with severe hypertriglyceridaemia, low HDL-C, hyperlipidaemias, non alcoholic fatty liver

low density lipoprotein
type 2 diabetes mellitus
fatty liver
LDL Cholesterol
statin
  • 215 views
  • 14 Jun, 2022
  • 1 location