Health Condition 1: Q688- Other specified congenital musculoskeletal deformities
Conditions
Interventions
Intervention1: NA: NA
Sponsors
Reliance life sciences
Eligibility
Inclusion criteria
Inclusion criteria: 1. Patients with known diagnosis of Duchenne Muscular Dystrophy (DMD) or Wilson’s disease 2. Patients who are = 5 years of age 3. Able to understand the study procedures, the risks involved, willing to provide written Informed Consent. For patients to provide written informed consent and subject should be willing to give assent for the study.
Exclusion criteria
Exclusion criteria: 4. Inability to comply to the protocol 5. Patients previously treated with any gene therapy for their disease. 6. Patients lacking mental capacity to consent to the trial 7. Seropositive for HIV/HBsAg / HCV (as per previous reports or test conducted prior to study enrolment is accepted) 8. Advanced stage of DMD or Wilson’s disease and / or other comorbidities that are likely influence the interpretation of the current study parameter.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To investigate the seroprevalence of AAV neutralizing factors and antibodies in patients of Duchenne Muscular Dystrophy and Wilsons disease.Timepoint: Day 0 | — |
Secondary
| Measure | Time frame |
|---|---|
| To collect the subject samples for biobanking & utilize these samples for the various evaluations / tests related to diagnosis or therapy of DMD & Wilson’s disease.Timepoint: Day 0 & future research.;To describe demographic & disease profile of patients of DMD & Wilson’s disease who are having preexisting anti AAV AntibodiesTimepoint: Day 0 | — |
Countries
India
Contacts
Public ContactMr Manoj Mavelil
Reliance Life Sciences Pvt. Ltd. (RLS)
Outcome results
None listed