Inclusion Criteria:
Inclusion Criteria For MLD:
1. Confirmed diagnosis as MLD by ARSA genetic diagnosis, MRI(Magnetic Resonance Imaging)and low ARSA A activity (below 20% of normal level);
2. The patient' symptoms and lesions have not been developed to the end stage of MLD.
3. age \< 16.0 years at symptom onset
Inclusion Criteria For ALD:
1. Confirmed diagnosis as ALD by ABCD1 genetic diagnosis, abnormal MRI imaging, abnormal high level of very long chain fatty acid (VLCFA) and adrenocorticotropic hormone (ACTH);
2. The patient' symptoms and lesions have not been developed to the end stage of ALD.
3. age \< 16.0 years at symptom onset
Exclusion Criteria:
Exclusion Criteria For MLD:
1. At a pre-symptomatic stage of of MLD;
2. ARSA activity \>50% compared to healthy individuals;
3. End stage of MLD;
4. Other complications, ie. Cancer;
5. human immunodeficiency virus(HIV) RNA and/or hepatitis C virus RNA and/or hepatitis B virus DNA positive patients;
6. Patients who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin.
7. Serious organ dysfunction;
8. were enrolled in other clinical trials in the 6 months prior to screening;
9. had any other concern that hampered the compliance or safety as judged by the investigator;
10. Adult
Exclusion Criteria For ALD:
1. No evidence of brain lesions;
2. Normal level of VLCFAs in blood;
3. End stage of ALD;
4. Other complications, ie. Cancer;
5. human immunodeficiency virus(HIV) RNA and/or hepatitis C virus RNA and/or hepatitis B virus DNA positive patients;
6. Patients who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin.
7. Serious organ dysfunction;
8. were enrolled in other clinical trials in the 6 months prior to screening;
9. had any other concern that hampered the compliance or safety as judged by the investigator;
10. Adult
View Inclusion and Exclusion Criteria at ClinicalTrials.gov