Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3

Noha Osman

Status: Invitation-only

Conditions: Gaucher Disease Type 3

Treatments: Diagnostic Test: echo

Study type: Observational

Funder types: Other

Identifiers

NCT06627543

04-2023-100034

Details and patient eligibility

About

all cases confirmed diagnosis of Gaucher disease type 3 recieving enzyme replacement therapy at Assiut university hospital

Full description

evaluate cardiac involvement in a group of GD type 3 patients, evaluate pulmonary hypertension and valvular involvement, compare the findings of basic echo before the start of replacement therapy as well as with healthy controls, to assess the effects of enzyme replacement therpay on the cardiac function.

Enrollment: 44 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Any sex
• No age limit
• Gaucher disease type 3
• Receiving enzyme replacement therapy

Exclusion criteria

• Age18 years old
• Cases of gaucher type 1 and 2
• Other lipid storage disease

Trial design

44 participants in 1 patient group

Gaucher type patients

Treatment:

Diagnostic Test: echo