Research Faces of MLD

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Research - Phases

Research and approval of a new therapy or drug to treat a disease is a very lengthy, expensive, and detailed process.

While some adjustments are made with rare diseases like MLD, the same general eight-step flow is followed. For example, adjustments may be made to the size of the test samples due to the relative rarity of the disease.

The general research-to-approval outline shown below is modeled on a real gene therapy research project from the University of Utah.

A detailed explanation of the details of the human Clinical Trials is here.


Eight Steps from Research to Approval

1) Learn about the disease to see if it is the disorder a good candidate for a new therapy

1) Get money for this preliminary study

2) Get approval for the project from your organization's Institutional Review Board

3) Perform clinical research - including publishing your results for peer review

4) Perform biological research - including publishing your results for peer review

2) Design a therapy to research

1) Use your knowledge of the disorder to design a gene therapy

2) Test the therapy in appropriate models of the disease

3) Obtain funding and approval for clinical trials

1) Get money for the trials

2) Get approval for the trials from your organization's Institutional Review Board

4) Phase One Clinical Trial details here

establish safety and dosage limits in a small group of people (20-80)

5) Phase Two Clinical Trial details here

test the efficacy and safety in a larger group of people (100-300)

6) Phase Three Clinical Trial details here

test the therapy in a large group of people (1,000-3,000)

7) Obtain FDA approval for general clinical use

8) Phase Four Clinical Trial details here

further test the efficacy and optimal use of the treatment in general use

 


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