Last updated Aug. 6, 2025, by Agata Boxe
Fact-checked by Ana de Barros, PhD
Although researchers have made progress developing therapies for spinal muscular atrophy (SMA), more work is needed to address disease progression and improve strength, mobility, and quality of life.
Scientists continue to explore new treatments through clinical trials, which assess whether new therapies are safe and effective before they are approved by the U.S. Food and Drug Administration (FDA).
Enrolling in an SMA clinical trial may give you or your child access to experimental treatments while helping to advance medical research. However, it is important that you consult your doctor or your loved one’s doctor as part of your decision-making process.
A clinical trial is a carefully designed research study that tests new ways to treat, diagnose, and manage diseases, such as SMA, in people. While early-stage clinical trials typically follow laboratory and animal testing, later-stage trials build on findings from earlier human studies.
Clinical trials for SMA may focus on:
Clinical trials come in four separate phases:
While the progressive nature of SMA and its severity may make someone more willing to join a clinical trial, it’s crucial to understand both the potential benefits and the risks.
The benefits of clinical trials include:
The risks of clinical trials can involve:
Clinical trials for SMA can focus on various aspects of SMA care, ranging from testing new interventions that modify the disease course or improve quality of life to identifying new biomarkers that aid in monitoring disease progression and response to treatment.
Not all trials may be available or recruiting participants at any given time, but some examples of SMA trials that may be conducted include:
Some SMA trials are tailored to people with specific SMA types as well as different age groups or genetic profiles. They may also be available only to individuals who have never received treatment or who are no longer responding to approved SMA treatments.
To find an SMA clinical trial, start by talking to your or your child’s SMA doctor, who may know of trials through academic hospitals, research centers, or clinical trial networks. They can also help evaluate whether you or your child may be eligible for a trial.
Additionally, you can consult online resources for more information. Resources that may help you find a suitable SMA trial in the United States include:
SMA trial registries outside the United States include:
If you’re considering participating in an SMA clinical trial, asking the right questions can help you make an informed decision. It may be helpful to put together a list of questions for your SMA doctor, the research team, and even yourself as you learn more about the trial process.
Your doctor can answer general questions about SMA trials, such as:
The research team running the trial can address more detailed questions about its design and potential side effects.
Examples of questions for the researchers include:
Inquiring about potential costs is especially important. Although most trials tend to cover the costs of experimental treatment and any related medical care, that may not be the case for every single study.
If you are currently insured, ask your insurer whether they can pay for your participation in the study and care for potential side effects.
Personal reflection can help you decide whether a trial might be right for you. Questions to ask yourself include:
SMA clinical trials can provide a chance to try experimental treatments and play an active role in your or your child’s SMA care. However, keep in mind that they’re not for everyone, so it’s important to understand their risks and benefits.
Remember to talk to your SMA doctor first, so they can help you decide whether an SMA trial is right for you. Don’t hesitate to ask questions and advocate for yourself as you make this and other key decisions about your health and future.
SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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