Clinical Trials

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:

  1. SMN1gene replacement
  2. Modulation of theSMN2 ‘back-up’ gene / antisense oligonucleotides (ASOs)
  3. Modulation of theSMN2 ‘back-up’ gene / small molecules
  4. Neuroprotection of motor neurons
  5. Muscle protection