SMA Research and Our Contribution
This takes place in the laboratory and is the first step in identifying and developing potential treatments and a possible cure for SMA.
Biomarkers are medical indicators that can be measured to inform us of progression of a disease. A dependable set of biomarkers for SMA will be an invaluable tool for assessing the effectiveness of potential treatments in SMA clinical trials.
We outline five different approaches that have reached human clinical trial stages and show promise in treating SMA - bearing in mind that approximately 9 out of 10 experimental drugs starting clinical trials never gain final approval.
They are not all old, bespectacled men with messy, white hair and a lab coat, totally disconnected from the outside world!
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