Clinical Trials for SMA

In these pages, we outline four different approaches that have reached human clinical trial stages and show promise in treating SMA – bearing in mind that 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.

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

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

1. SMN1 Gene Replacement2. SMN2 Gene Enhancement3. Motor Neuron Protection4. Muscle Protection
Novartis Gene Therapies (AveXis)
SRK 015
Scholar Rock
Risdiplam (RG7916)


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