Clinical Trials for SMA

What is a clinical trial?

A clinical trial is a rigorously controlled test designed to examine the safety and/or effectiveness of medicines, devices, treatments, or preventive measures in humans. Clinical trials follow a strict protocol. The protocol describes the aims, design and organisation of a clinical trial. It also provides information on the background and reasons for atrial and outlines the study plan for that trial. The plan must be carefully designed to ensure the safety of the participants as well as answer specific research question(s).

Clinical trials for SMA

The identification, development and approval of drugs is a long, costly, and difficult process. It is estimated that 9 out of 10 experimental drugs starting human clinical trials never gain final approval.

Researchers have identified four main different therapeutic approaches that show promise in treating SMA:

SMN1 gene replacement

Modulation of the SMN2 ‘back-up’ gene

Neuroprotection of motor neurons

Muscle protection

For a very useful overview, please read this document created by the SMA Foundation in the US and endorsed by SMA Europe, by clicking on the image below:

Clinical Trials for SMA


Clinical Trials for SMA