The Importance of Clinical Trials in SMA

Cure SMA has released a booklet highlighting the importance of clinical trials for research into better treatments and therapies for spinal muscular atrophy (SMA) patients.

MORE: Phase 2 clinical trial of oral compound targeting genetic cause of SMA starts in Europe.

The brochure explains the processes involved in clinical trials, what to expect if you or your child participates in one, and how the drugs or therapies in clinical trials are not guaranteed to improve SMA symptoms or slow disease progression.

There’s an outline of the four phases of a clinical trial and how long each phase typically takes. Bringing a new drug to the FDA for approval can take between 10 to 15 years. Some clinical trials are fast-tracked if initial data shows promising results and major improvements over current treatments.

The other thing to note is that not all participants in a clinical trial will receive the new medication. Half of the participants will be given a placebo (or sham) and will be used as part of a control group so researchers can compare patients taking the new drug against those not taking it.

MORE: Nusinersen found safe and effective in treating infants with type 1 SMA.

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