Clinical trials
Spinal Muscular Atrophy clinical trials
Below are recruiting spinal muscular atrophy clinical trials, each written for real people, not researchers. We’re tracking 63 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06756633Recruiting
Breathing and movement study for neuromuscular disease
This study looks at breathing, chest and belly movement, and exercise ability in people with Duchenne Muscular Dystrophy or Spinal Muscular Atrophy, as well as healthy people. The goal is to compare these functions to help understand how these diseases affect the body.
Ankara, ÇankayaAges 5–15 - NCT06839469Recruiting
Walking study for rare muscle and nerve conditions
This study looks at how children and teens with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk, by having them do walking tests while wearing sensors. The goal is to find better ways to track how these diseases progress and how treatments work over time, without needing blood tests or scans.
Palo Alto, CaliforniaAges 5 years+ - NCT06862596RecruitingPhase 2/Phase 3
Mexiletine for spinal and bulbar muscular atrophy
This trial tests whether mexiletine hydrochloride can help men with spinal and bulbar muscular atrophy (SBMA) by improving muscle strength and function. It may be an option if you have mild to moderate symptoms and meet heart and medication safety checks.
Bunkyō CityAges 18–80 - NCT06971094RecruitingPhase 3
Gene therapy for type 2 spinal muscular atrophy
This trial tests a new gene therapy called GC101 given as a one-time injection into the spinal fluid for people with type 2 spinal muscular atrophy (SMA). It may help improve motor function.
BeijingAges 2–12 - NCT06977269Recruiting
Brain stimulation safety study for spinal muscular atrophy
This trial tests whether a non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) is safe and tolerable for people with spinal muscular atrophy (SMA). It may help improve muscle function by activating low motor neurons.
Kharkiv, Kharkiv OblastAges 12 years+ - NCT07047144RecruitingPhase 2
Apitegromab for infants with SMA under 2 years old
This study tests a new medicine called apitegromab for babies under 2 years old with spinal muscular atrophy (SMA). It looks at whether the drug can help with muscle strength and reaching motor milestones like sitting or rolling.
Phoenix, ArizonaAges Up to 2 years - NCT07070999RecruitingPhase 1/Phase 2
Gene therapy GB221 for infants with SMA type 1
This trial tests a new gene therapy called GB221 for infants with SMA type 1, a rare muscle-weakening disease. It aims to see if this treatment can improve muscle strength and development, and it is for babies who have not had prior gene therapy.
Porto Alegre, Rio Grande do SulAges 2 weeks–1 year - NCT07223489Recruiting
Understanding Adult SMA Diagnosis & Treatment Experiences
This trial explores the experiences of adults with spinal muscular atrophy (SMA) in the MedStar Health System, looking at how they are diagnosed and treated, and any differences in care they may face.
Washinton, District of ColumbiaAges 18 years+ - NCT07231549Recruiting
SMA study tracking health over time in Spain
This study collects health information from people with spinal muscular atrophy (SMA) over time to learn more about the disease and how to improve care. It may help researchers understand SMA better.
Esplugues de LlobregatAges Any age - NCT07265232RecruitingPhase 3
Gene therapy for spinal muscular atrophy in low-resource settings
This trial tests a new gene therapy called Vesemnogene Lantuparvovec for people with spinal muscular atrophy (SMA). It is designed for patients in low- and middle-income countries who cannot access or did not respond to other treatments. The goal is to see if the therapy is safe and effective in real-world conditions.
Jakarta, IndonesiaAges 6 months+ - NCT07286565Recruiting
Remote motor development study for early-diagnosed DMD or SMA
This study remotely tracks motor development in children with Duchenne muscular dystrophy or spinal muscular atrophy who were diagnosed at birth or before symptoms started. It aims to see if early monitoring helps improve long-term care and outcomes.
LiègeAges 4 months+ - NCT07332702Recruiting
Spinal muscular atrophy gene study
This study looks at changes in two genes, SMN1 and SMN2, which are linked to spinal muscular atrophy (SMA). It aims to better understand the different versions of these genes in healthy people and those with SMA. Participating involves a blood sample and genetic analysis.
RouenAges 18 years+ - NCT07400198Recruiting
Walking and Bone Health Study for SMA
This study looks at walking and bone health in people with spinal muscular atrophy (SMA). It helps researchers understand how SMA affects your gait (the way you walk) and your bones, which could lead to better treatments.
New York, New YorkAges 8–50 - NCT07444476RecruitingPhase 3
Study of salanersen for spinal muscular atrophy (ages 15-60)
This trial tests a medicine called salanersen (BIIB115) to see how well it improves movement and how safe it is in people with spinal muscular atrophy (SMA). It includes those who have never taken an SMA drug or are currently on risdiplam and willing to switch.
Norfolk, VirginiaAges 15–60 - NCT07467187Recruiting
Home Ventilation with Tracheostomy in Denmark (2016–2025)
This study looks at people in Denmark who need a home ventilator through a tracheostomy. It aims to understand their care and outcomes over several years.
CopenhagenAges Any age - NCT07478172Recruiting
Electrical muscle stimulation exercise for neuromuscular disease
This study tests whether whole-body electrical muscle stimulation (using small electrodes on the skin) can help adults with certain neuromuscular diseases exercise and improve muscle strength. It may be an option if you can stand for about 15 minutes at a time and have some muscle strength left.
Columbia, MissouriAges 18 years+ - NCT07543003Recruiting
Wearable Monitoring for Young Children With Spinal Muscular Atrophy
This study uses wearable devices (like smartwatches or activity trackers) to monitor movement and motor development in young children with spinal muscular atrophy (SMA), a rare genetic condition affecting muscle strength. The goal is to see if wearables can track disease progression and treatment response better than traditional clinic visits.
OxfordAges Up to 4 years - NCT06888661Enrolling by invitationEarly Phase 1
Testing EXG001-307 for spinal muscular atrophy in infants
This trial tests a new medicine called EXG001-307 for babies with spinal muscular atrophy (SMA) types 1 and 2. The goal is to see if it is safe and helps with muscle strength and breathing.
Shanghai, Shanghai MunicipalityAges 1 day–2 years - NCT06772402Enrolling by invitation
Gene therapy for children with type 2 SMA
This trial tests a new gene therapy called GCB-001 for children with type 2 spinal muscular atrophy who can sit but not walk. It aims to improve muscle function and overall health.
Hangzhou, ZhejiangAges 2–12 - NCT05614531Enrolling by invitationPhase 1/Phase 2
Safety and benefit study of EXG001-307 for baby spinal muscular atrophy
This Phase 1/2 study tests whether EXG001-307 is safe and may improve health in babies with spinal muscular atrophy (SMA) type 1. It mainly looks at safety first, and whether the treatment can help the body work better over time.
Shanghai, Shanghai MunicipalityAges 1 day–6 months - NCT07521930Recruiting
Brain implant to restore communication and movement control
This trial tests a surgically implanted brain device designed to help people with severe paralysis or movement disorders regain the ability to communicate and control their limbs. The device reads signals from your brain and translates them into commands that could help you interact with computers or assistive technology.
Baltimore, MarylandAges 18–80 - NCT07321977Recruiting
Walking test with a portable device for neuromuscular disease
This study tests a portable device that analyzes walking without markers. It aims to see if it can help assess walking in people with neuromuscular diseases and healthy volunteers, which could lead to simpler check-ups.
ParisAges 18–65 - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - NCT07072676Enrolling by invitation
Can a walker help prevent falls in neuromuscular disease?
This study tests if using assistive walking devices, after a short training period, can lower the risk of falls in people with neuromuscular diseases. It aims to help you stay steady and safe on your feet.
Bad FeilnbachAges 65–65
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Common questions
- Are there clinical trials for spinal muscular atrophy?
- Yes. Clin2 currently lists 63 recruiting spinal muscular atrophy studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a spinal muscular atrophy trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a spinal muscular atrophy trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.