FREE PATIENT NAVIGATOR SUPPORT

See which spinal muscular atrophy clinical trials may be right for your child 

If you are wondering whether clinical trials might be an option for your child, you are not alone. Our patient navigators offer free, no-obligation guidance to help you explore SMA trial options. 

Find trials for my child 

New to clinical trials? Start with the basics

· Trusted by 8,000+ NMD patients 
· Free 15-30 min call in 15+ languages  
· Your information is kept private

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Wherever you are in your search,
a patient navigator can help 

Whether your child is on treatment and you’re wondering what comes next, was recently diagnosed, or you’re only starting to explore — a patient navigator can help you find SMA clinical trial options that may be relevant for your child. We have supported families in the U.S. and over 135 other countries. 

A specific SMA trial you've found

Walk through the details, requirements, and what to ask the trial team. Bring any trial name, NCT number, or link you've come across. 

On treatment, exploring what's next

If your child is on Spinraza or Evrysdi, or progress has slowed or plateaued, a navigator can help you explore SMA trials that may be relevant.

After gene therapy

If your child has had Zolgensma or is past the age it's given, a navigator can help you explore relevant SMA trials, including studies for children who've already had gene therapy. 

Your child was recently diagnosed

Whether through newborn screening or a recent diagnosis, a navigator can usually speak with you soon to talk through which SMA trial options may be relevant. No need to have everything ready to start. 

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Why families work with a navigator 

Information about SMA clinical trials is scattered, fast-changing, and often hard to interpret without specialist knowledge. New trials open, others close, and eligibility criteria can shift month to month. For SMA specifically, whether a trial may be relevant depends on several factors at once — your child’s age, SMA type, functional status, SMN2 copy number, and treatment history. Even engaged, well-informed families lose time and miss options trying to make sense of it all alone. 

A patient navigator does this work full-time. They keep up with the SMA research landscape as it changes, match what's open to your child's specific situation, and explain what each option may involve so you can make decisions with clarity instead of guesswork. 

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Understanding clinical trials for spinal muscular atrophy 

Clinical trials are research studies that test new treatments to see if they are safe and how well they work. For SMA, trials are studying several new approaches: muscle-directed therapies that target muscle strength alongside the underlying disease, new gene therapy options, improved ways to deliver existing treatments, and combinations of treatments. Others look at ways to slow progression or help maintain function over time. These treatments are investigational, which means researchers are still studying them and they are not yet approved. 

Why families consider trials

Possible access to investigational treatments not yet available 

Regular check-ups and care from the clinical trial team

Contributing to SMA research that may benefit future families

Important to know: Clinical trials aren't right for everyone, come with risks, and don't guarantee benefit. A call with a navigator can help you understand whether a trial may make sense for your child and what questions to ask before deciding anything.

How we help

Your personal patient navigator

Our patient navigators are SMA trial experts. They review your child’s age, SMA type, functional status, SMN2 copy number, and treatment history to check them against an up-to-date list of SMA trials. 

Step 1

Book your call

Fill out a short form and select a call time. It takes just a few minutes and your information stays private. 

Step 2

Speak to your navigator

During your free 15–30 minute call, your navigator learns about your child’s situation, answers your questions around eligibility, and discusses which SMA trials may be worth exploring further.

Step 3

Explore trials together

After the call, you’ll receive a personalized overview of potentially relevant SMA trials. If a trial interests you, a navigator can help with next steps, including connecting with trial sites in the U.S. or internationally. You stay in control. 

Free

15+ languages

No obligation

Independent

Not medical advice

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Why SMA families trust myTomorrows

Since 2012, we have supported thousands of families across 135+ countries in navigating rare disease clinical trials — including thousands of families affected by neuromuscular diseases. We are independent, which means our navigators provide unbiased support focused on your child's unique situation.

8,000+

Neuromuscular disease patients supported worldwide

135+

Countries

13+

Years of rare disease expertise

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Frequently asked questions (FAQs)

Is this relevant for my child with SMA? 

If your child has been diagnosed with SMA — whether as a newborn, infant, or older child — there may be clinical trial options to explore, depending on their age, SMA type, functional status, SMN2 copy number, and treatment history. These factors can be hard to sort through alone, but a short call with a patient navigator can help you understand which options may be relevant and what to keep an eye on as new trials open. You can explore options without committing to anything. 

My child is already on Spinraza or Evrysdi, can they still explore SMA clinical trials? 

Yes, in many cases. Some SMA clinical trials are specifically designed for children who are already receiving Spinraza or Evrysdi, while others have different requirements around prior treatment. A patient navigator can help you understand which options may be relevant given your child’s current treatment and how they have been responding to treatment. The clinical trial team confirms the final eligibility details for any specific trial. 

My child’s newborn screen came back positive for SMA, what should I do? 

The most important first step is to connect with your child's medical team or an SMA specialist if you haven't already — they can guide your child's immediate care. At the same time, a patient navigator can help you understand which SMA clinical trial options may be relevant alongside whatever treatment path your team recommends. You don't need to wait, and you don't need to have everything ready to start. 

My child has had Zolgensma, are there still SMA clinical trial options to explore? 

Yes. Having had Zolgensma does not rule out exploring SMA clinical trial options. Some trials are specifically designed for children who have already had gene therapy, studying approaches that may complement their current care. A patient navigator can help you understand which options may be relevant for your child now — and you can take the information and decide what feels right for your family. 

What happens if I book a navigator call? 

You'll speak with a patient navigator who will ask a few questions about your child's diagnosis, SMA type, treatment history, and current situation so they can understand which SMA clinical trial options may be relevant. If you have genetic results or SMN2 copy number information, it helps to share them — but you don't need everything ready to start. After the call, we'll explain what options may be a match, if any, and what next steps could look like. We won't contact any clinical trial team without your permission. 

Is this a commitment to join a clinical trial? 

Booking a call is not a commitment to joining a clinical trial. Many families speak with a patient navigator simply to understand what options may exist and what joining an SMA trial could involve. After the call, you can decide what, if anything, you want to do next — including just staying informed. Any next step would only happen after speaking with your child's doctor and the clinical trial team, who can give you the full details of the trial. 

What if my child is not eligible for an SMA clinical trial? 

That can happen and it is common. SMA clinical trials often have specific requirements related to age, SMA type, functional status, SMN2 copy number, treatment history, or other medical details. If your child is not eligible right now, your navigator can explain why, help you understand what to watch for, and keep you informed as new trials open or criteria change. 

Do I need genetic results, including SMN2 copy number, to explore SMA clinical trials? 

Not necessarily, but they are often helpful. Many SMA clinical trials have specific criteria related to genetic results, including SMN2 copy number, so having those details can give the navigator a clearer picture of which options may be relevant for your child. If you already have genetic results, you can share them during or after the call — if you don't, your navigator can explain what information may be useful and what to ask your child's care team to gather. We'll only share information with a clinical trial team if you ask us to. 

Will we need to travel for an SMA clinical trial? 

Sometimes, yes. Many SMA clinical trials take place at specialized hospitals, and joining a trial may require in-person visits for tests, check-ups, or treatment. How much travel is needed depends on the specific trial — some have sites in multiple regions, while others are only available in a few locations. A navigator can help you understand what joining a trial could involve, including where sites are located and what the visit schedule may look like, and the clinical trial team can confirm practical details including any travel support. 

Is there any cost to speak with a patient navigator? 

No, there is no cost to speak with a patient navigator. The call is for information and guidance so you can understand which SMA clinical trial options may be relevant and what next steps could look like. You can take that information and decide what feels right for your family. 

Will my information be kept private? 

Yes. We handle any medical information you share securely and use it only to assess whether SMA clinical trial options may be relevant for your child. We will not share your information with any clinical trial team or hospital without your clear permission. You can ask questions, choose what you want to share, and decide what happens next. 

More on clinical trials

What is a clinical trial?

A clinical trial is a research study that tests a new treatment or approach to see how safe it is and how well it works to treat a medical condition. Clinical trials follow strict rules and are reviewed by independent ethics committees to help protect participants. What’s involved can vary by trial, including clinic visits, tests, and how the treatment is given. If you’re exploring options for SMA, a navigator can help you understand what clinical trial options may be relevant and what joining a trial could involve. Joining a clinical trial is always voluntary. 

Why do families consider clinical trials for SMA?

Families consider SMA clinical trials for different reasons. Some want to learn about new treatment approaches — especially if they are wondering what other options may be available alongside their child's current care, or what comes next. Others value the regular check-ups and specialist follow-up that can come with joining a trial, or want to support research that may help other SMA families in the future. A patient navigator can help you understand what options may be relevant and what joining a trial could involve, so you can discuss it with your child's care team.

Are SMA clinical trials safe for children?

SMA clinical trials follow strict safety rules, but there can still be risks and side effects. Before a child can join a trial, the clinical trial team reviews medical information to check whether the trial is appropriate. During the clinical trial, children are monitored closely through regular visits and tests. Clinical trials are also reviewed by independent ethics committees to help protect participants. If you’re exploring options, a navigator can explain what safety monitoring often looks like and what questions to ask a clinical trial team. Joining a clinical trial is always your choice, and you can stop at any time.

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Talk to a patient navigator

Fill out this short form to book your free call.
You can select your time and language on the next page

What to expect:

A free 15-30 min call with a patient navigator who specializes in SMA trials

Review of your child's diagnosis and current care

A clear plan for finding SMA trials that may match

No obligation. Your information is never shared without your permission

Important: If you are a caregiver, please note that the patient’s legal guardian needs to be present on the call. A legal guardian is someone who has the legal right to make decisions for the patient, such as a parent of a minor or a court-appointed representative. 

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