Condition

Spinal Muscular Atrophy clinical trials and research

• 19Clinical trials
• 13NIH research projects
• 15Countries

Newest Spinal Muscular Atrophy clinical trials

All recruiting trials (19)

• Pregnancy-time treatment of fetal SMA: a registry of mother-child outcomes

  This registry collects information on pregnancies where a fetus diagnosed with spinal muscular atrophy was treated with a disease‑modifying therapy in the womb to see how the pregnancy and baby fare after birth.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT07805278

• Intrathecal Zolgensma in spinal muscular atrophy patients in Japan

  This study tests the safety and real‑world effectiveness of intrathecal onasemnogene abeparvovec in SMA patients in Japan.

  Recruiting at 4 sites Novartis · Toyoake, Aichi-ken and 3 other locations · NCT07767890

• Spinal muscular atrophy patients get a single intrathecal gene therapy dose to see how it works in real-world U.S. settings

  This study tests whether a one-time intrathecal dose of ITVISMA® (onasemnogene abeparvovec-brve) is safe and effective for SMA patients in real-world U.S. clinical practice.

  Recruiting at 6 sites Phase 4 Novartis · Little Rock, Arkansas and 5 other locations · NCT07448610

• Salanersen (BIIB115) for improving movement in people 15–60 with spinal muscular atrophy who are treatment‑naïve or previously on risdiplam

  This trial tests whether salanersen helps movement and is safe for people aged 15 to 60 with spinal muscular atrophy who are either new to treatment or have taken risdiplam.

  Recruiting at 3 sites Phase 3 Biogen · Palo Alto, California and 2 other locations · NCT07444476

• CuidAME: Spain's longitudinal registry for spinal muscular atrophy

  This project collects long-term clinical and treatment data from children and adults with genetically confirmed 5q spinal muscular atrophy in Spain to track outcomes over time.

  Recruiting Fundació Sant Joan de Déu · Esplugues de Llobregat · NCT07231549

• Apitegromab for infants under 2 with spinal muscular atrophy

  This trial will test whether adding apitegromab to approved SMA therapy helps infants under 2 with 5q SMA reach better motor milestones.

  Recruiting at 23 sites Phase 2 Scholar Rock, Inc. · Phoenix, Arizona and 22 other locations · NCT07047144

• Understanding fatigue in patients with spinal muscular atrophy treated with nusinersen

  This study is trying to see how nusinersen treatment affects fatigue in people with spinal muscular atrophy and how that fatigue impacts their daily activities.

  Recruiting Columbia University · New York, New York · NCT06955897

• Evaluating respiratory functions and exercise capacity in children with neuromuscular diseases

  This study is trying to see how well children with Duchenne Muscular Dystrophy and Spinal Muscular Atrophy can breathe and exercise compared to healthy kids.

  Recruiting Lokman Hekim University · Ankara, Çankaya · NCT06756633

• Examining muscle responses in spinal muscular atrophy patients treated with specific therapies

  This study is testing how different treatments for spinal muscular atrophy affect muscle and nerve responses in patients over a year.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT06532474

• Home-based exergaming for children with spinal muscular atrophy

  This study is testing if playing a fun video game at home can help kids and teens with spinal muscular atrophy feel happier and more engaged in their daily activities.

  Recruiting at 5 sites McGill University Health Centre/Research Institute of the McGill University Health Centre · Calgary, Alberta and 4 other locations · NCT06396325

• Characterizing new patient types with spinal muscular atrophy treated with SMN therapies

  This study is trying to understand how different types of spinal muscular atrophy in children under 16 respond to new treatments that boost SMN protein levels, while also looking at their muscle strength, thinking skills, and heart health over two years.

  Recruiting Hospices Civils de Lyon · Bron, Rhone and 7 other locations · NCT06321965

• Implantation of a device for delivering nusinersen in spinal muscular atrophy patients

  This study is testing a new device that delivers a treatment for spinal muscular atrophy directly into the spine to see if it is safe and works well for patients who have trouble with regular injections.

  Recruiting at 21 sites Alcyone Therapeutics, Inc · Phoenix, Arizona and 22 other locations · NCT05866419

• Gene therapy for treating spinal muscular atrophy Type 1

  This study is testing a new gene therapy for babies with spinal muscular atrophy Type 1 to see if it helps them sit up on their own by 18 months old.

  Recruiting at 4 sites Phase 1/2 GeneCradle Inc · Beijing and 3 other locations · NCT05824169

• Understanding the long-term effects of treatments on Spinal Muscular Atrophy patients

  This study looks at how different treatments affect people with Spinal Muscular Atrophy over time, comparing those who are treated and those who aren't to understand their quality of life and disease progression.

  Recruiting at 5 sites Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Genova and 4 other locations · NCT05768048

• Identifying biomarkers for early diagnosis of motor neuron diseases

  This study is trying to find new ways to diagnose motor neuron diseases early by looking at brain and muscle signals in patients to help improve their care.

  Recruiting University of Dublin, Trinity College · Dublin, Leinster · NCT05663008

• Registry for patients with Spinal Muscular Atrophy in the UK

  This study is collecting information from people with Spinal Muscular Atrophy to better understand their experiences and improve future treatments.

  Recruiting Newcastle University · Newcastle upon Tyne · NCT04292574

• Registry of Patients with Spinal Muscular Atrophy in France

  This study is collecting health information from people with spinal muscular atrophy in France to see how different treatments affect their survival and overall well-being.

  Recruiting Assistance Publique - Hôpitaux de Paris · Garches · NCT04177134

• Cohort study on spinal muscular atrophy in China

  This study is trying to learn more about spinal muscular atrophy by looking at how it affects different patients in China and how their genes relate to their symptoms.

  Recruiting First Affiliated Hospital of Fujian Medical University · Fuzhou, Fujian · NCT04010604

• Screening for Spinal Muscular Atrophy in Newborns

  This study is testing a new way to screen newborns in Taiwan for Spinal Muscular Atrophy to catch it early and help affected babies get the care they need.

  Recruiting Kaohsiung Medical University Chung-Ho Memorial Hospital · Kaohsiung City · NCT03217578

NIH-funded research (13)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Genetic clues to mouth muscles development in SMA

  A small NIH grant studies how nerve cells controlling jaw movements develop normally and how this process goes wrong in spinal muscular atrophy (SMA).

  NIH research Columbia University Health Sciences · New York, United States · NIH-11356582

• Therapeutic gene editing for spinal muscular atrophy

  A patient-facing look at a project that aims to test a gene-editing approach to help children with spinal muscular atrophy.

  NIH research Jackson Laboratory · Bar Harbor, United States · NIH-11326247

• SMN-independent approaches for spinal muscular atrophy

  This project looks for new treatments for people with spinal muscular atrophy that work without relying only on restoring the SMN protein.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11307153

• How RNA problems cause nerve and muscle damage in spinal muscular atrophy

  This project looks at how RNA-related problems lead to nerve and muscle damage in people with spinal muscular atrophy to help find better treatment targets.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11285173

• Investigating speech development in children with spinal muscular atrophy after new therapies

  This study is looking at how new treatments for spinal muscular atrophy (SMA) can help improve speech and communication skills in children and young adults aged 2 to 21, by comparing their speech abilities to what’s expected for their age.

  NIH research University of Wisconsin-Madison · Madison, United States · NIH-11242987

• Correcting Genetic Errors for Spinal Muscular Atrophy

  This research looks for new ways to fix the genetic problem that causes Spinal Muscular Atrophy, a condition that weakens muscles.

  NIH research Ohio State University · Columbus, UNITED STATES · NIH-11123252

• Gene editing to treat spinal muscular atrophy

  This study is working on a new gene editing treatment for spinal muscular atrophy (SMA) to see how well it can help improve muscle strength and overall health, especially for infants affected by this condition.

  NIH research Jackson Laboratory · Bar Harbor, United States · NIH-11089352

• Investigating the effects of antisense oligonucleotides on spinal muscular atrophy treatment

  This study is looking at a new way to help people with spinal muscular atrophy (SMA) by testing special treatments that could improve how a key gene works, with the hope of finding better therapies to make life easier for SMA patients.

  NIH research Iowa State University · Ames, United States · NIH-11078218

• Investigating new therapies for spinal muscular atrophy

  This study is looking at new ways to help people with spinal muscular atrophy (SMA) by exploring treatments that go beyond just fixing the SMN protein, aiming to find better options for young patients with this condition.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11059216

• Creating a lasting genetic treatment for spinal muscular atrophy

  This study is testing a new one-time genetic treatment for spinal muscular atrophy (SMA) that aims to help improve the lives of people with this condition by making precise changes to a key gene.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11012259

• Investigating how calcium regulation affects heart function in spinal muscular atrophy.

  This study is looking at how a lack of a certain protein affects the heart in children with spinal muscular atrophy (SMA), hoping to find new ways to help manage heart problems that can come with this condition.

  NIH research Henry M. Jackson Fdn for the Adv Mil/med · Bethesda, United States · NIH-11005051

• Finding ways to improve SMN protein levels in spinal muscular atrophy

  This study is looking at ways to improve a protein that's important for muscle health in people with spinal muscular atrophy (SMA) by testing new treatments that could help fix problems caused by certain gene mutations.

  NIH research Ohio State University · Columbus, UNITED STATES · NIH-10884302

• Investigating brain changes in spinal muscular atrophy

  This study is looking at how spinal muscular atrophy (SMA) affects a part of the brain called the cerebellum, which helps with movement, using special imaging techniques and mouse models to find out more about the brain changes in SMA, with the hope of improving treatments for people with this condition.

  NIH research Delaware State University · Dover, United States · NIH-10436518

Related topics

Quick answers

How many Spinal Muscular Atrophy clinical trials are recruiting right now?

19, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 15 countries. The most sites are in United States (7 trials), China (3 trials), United Kingdom (3 trials), France (3 trials), Spain (3 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1/2: 1, Phase 3: 1, Phase 4: 1, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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