Clinical Trials for SMA

In these pages, we outline 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 different therapeutic approaches that show promise in treating SMA:

Novartis Gene Therapies
Spinraza (nusinersen) - BiogenRisdiplam (Evrysdi) - RocheApitegromab (SRK-015)
Scholar Rock
Mechanism of actionIncreases SMNIncreases SMNIncreases SMNSMN independentSMN independent
StrategySMN1 gene replacementSMN2 splicing modifier (gene enhancement)SMN2 splicing modifier (gene enhancement)Muscle enhancementMuscle enhancement
Drug typeGene therapyASOSmall moleculeAntibodyFast skeletal muscle troponin activator
Delivery methodIVITOralIVOral


Clinical trials for SMA