Clinical trials
SMA clinical trials
Below are recruiting sma clinical trials, each written for real people, not researchers. We’re tracking 49 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05755451Recruiting
Study of the natural course of spinal muscular atrophy
This study observes how spinal muscular atrophy changes over time without giving an investigational treatment. It may help researchers learn what to expect in the disease and improve future care.
GenovaAges Any age - NCT05768048Recruiting
Study tracks SMA patients with specific gene changes
This study follows people with spinal muscular atrophy (SMA) who have a particular genetic change in the SMN1 gene. It compares long-term outcomes for those who do and do not receive disease-modifying treatments, to better understand what helps over time.
GenovaAges Any age - NCT05789758Recruiting
Study of Spinraza in pregnancy for 5q spinal muscular atrophy
This study looks at how Spinraza (nusinersen) taken during or before pregnancy affects people with 5q spinal muscular atrophy (SMA) and the health of their babies. It mainly fits those who were pregnant and received Spinraza around the time of conception or during pregnancy.
Palo Alto, CaliforniaAges 18–54 - NCT05824169RecruitingPhase 1/Phase 2
Gene therapy for babies with severe spinal muscular atrophy
This early-stage study tests whether a one-time gene therapy can safely improve outcomes for babies with the most severe type of spinal muscular atrophy (SMA type 1). It focuses on safety and early signs of benefit, and participation requires specific genetic and health criteria.
BeijingAges birth–6 months - NCT05852418Recruiting
Registry study of devices, medicines, and care in ALS and SMA
This study collects information in a registry about assistive devices, medicines, and healthcare support for people with long-term neurological conditions like ALS and SMA. It may help researchers understand what treatments and supports are used and how they relate to patients’ needs over time.
BerlinAges 18 years+ - NCT06152302Recruiting
Water mobility test for infants with SMA
This study tests how well infants with spinal muscular atrophy (SMA) can move in water. The goal is to see if gentle water exercises can help with muscle weakness and overall development.
GarchesAges Up to 1 year - NCT06194539Recruiting
Fertility in Men with Spinal Muscular Atrophy
This study looks at fertility (the ability to have children) in men with spinal muscular atrophy (SMA). It will help doctors understand how SMA affects male fertility by tracking sperm health and related factors.
Cresskill, New JerseyAges 18–50 - NCT06363357Recruiting
Shoulder brace study for arm weakness from nerve or muscle diseases
This study tests a fabric shoulder brace that moves like a muscle. It may help people with nerve or muscle diseases improve arm movements like reaching and lifting.
Seoul, Jongno-guAges 10 years+ - NCT06421831RecruitingPhase 1/Phase 2
Gene therapy trial for spinal muscular atrophy type 3
This trial is testing a one-time gene therapy drug for people with SMA type 3. The goal is to see if it can safely improve muscle function and strength.
Beijing, Beijing MunicipalityAges 2 years+ - NCT06645587Recruiting
Studying brain stimulation to reduce repetitive behaviors in autism
This trial tests if a gentle brain stimulation technique called transcranial direct current stimulation (tDCS) can help children with autism who have repetitive behaviors. It is for children aged 8 to 13 with an IQ of 70 or higher.
Rome, ItalyAges 8–13 - NCT06666816Recruiting
Watching changes in walking for muscle and nerve diseases
This study watches how people with certain muscle or nerve diseases walk over time. It may help doctors understand changes in walking patterns and is open to adults who can walk and have a genetic diagnosis of a neuromuscular condition.
Bosisio Parini, LeccoAges 18–75 - NCT06955897Recruiting
Study of Fatigue in SMA Patients on Nusinersen
This study looks at how tired you feel during physical activities while being treated with nusinersen (Spinraza) for spinal muscular atrophy (SMA). The goal is to understand how well the treatment helps with daily energy levels.
New York, New YorkAges 12 years+ - 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 - 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+ - 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 - 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 - 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 - 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 - 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 - 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 - NCT06809387Enrolling by invitation
Magnetic brain stimulation for stuttering and reading in adults
This study tests whether a gentle magnetic stimulation technique called rTMS can help improve speech and reading fluency in adults who have stuttered since childhood. It aims to understand how a brain area involved in movement planning might play a role in stuttering.
IstanbulAges 18 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
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Common questions
- Are there clinical trials for sma?
- Yes. Clin2 currently lists 49 recruiting sma 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 sma 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 sma 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.