Cure SMA Releases New Clinical Trial Care Series Booklet

As more and more SMA drug programs progress through clinical trials, there is an increasing need for accurate and unbiased information on clinical trials from our community. Cure SMA has been working to address this through education efforts targeted to the specific needs our community.

The centerpiece of these education efforts is our new care series booklet, Learning About Clinical Trials. Developed with a panel of expert reviewers, many of whom are members of our Medical Advisory Council, this booklet introduces and explains the clinical trial process.

While a number of general resources on clinical trials exist, these do not address the unique challenges and opportunities of SMA. Importantly, our booklet fills the unmet need for clinical trial information that directly addresses SMA and the common questions in our community.

Unique Challenges and Opportunities

These crucial issues include:

  • The SMA treatments currently in development work on a number of different therapeutic approaches. Some target the underlying genetics, and some would protect nerves or muscles in order to slow or stop the disease. With these different approaches being tested on different ages, stages, and types of SMA, researchers face complex decisions in designing and evaluating clinical trials—decisions such as who is included in trials and how change is measured.
  • SMA is considered an “orphan disease.” For orphan diseases, the number of patients enrolled in each clinical trial phase is often smaller than the numbers for non-orphan diseases. Orphan diseases are also eligible for special incentives from the FDA to encourage or speed up drug development.
  • Because many of those affected by SMA are very young, SMA trials must consider additional factors when testing on infants or children.
  • Because SMA is a life-threatening disease that does not yet have an approved treatment, the question of expanded access is more critical than it might be for other diseases.

This care series booklet is now available online, and will be part of a clinical trial information packet that will be distributed at our Annual SMA Conference. Throughout the remainder of this year, we’ll continue to release new materials to aid the entire community.

Ongoing Educational Efforts

In addition to these education efforts, here are a few of the other ways we’re working with our community on clinical trials:

Have a question about clinical trials or a suggestion of other information that might be helpful? Send us an email at [email protected].

Do you like what you're reading?

Help make a difference in the lives of people affected by spinal muscular atrophy.

Leave a Comment

Your email address will not be published. Required fields are marked *

Scroll to Top