Spinal muscular atrophy
An inherited condition in which the nerve cells that control movement are lost, so the muscles become weak and waste away. There is no cure yet, but medicines that target the altered genes, and other care, can help. Symptoms usually start in babies and toddlers, but can start in teenagers or adults. It usually happens when both parents carry an altered gene but do not have the condition themselves.
Read more about spinal muscular atrophyTrial-stage treatments and research studies you can join
Filters
- Tests an implanted port for giving nusinersen to people with spinal muscular atrophy
Tests a port and catheter device for delivering nusinersen in people with spinal muscular atrophy.
Austin, United StatesAlso Baltimore, Boston, Charlottesville, Chicago, Cleveland, Grand Rapids, Hershey, Houston, …Age 3+ - Real-world safety and effectiveness of intrathecal onasemnogene abeparvovec in spinal muscular atrophy
Studies how well onasemnogene abeparvovec-brve works and how safe it is in spinal muscular atrophy.
Boston, United StatesAlso Columbus, Little Rock, New York, Philadelphia, StanfordAge 2 to 100 - Tests salanersen in babies with spinal muscular atrophy before symptoms appear
Tests salanersen in newborn babies with genetic spinal muscular atrophy and no symptoms yet.
Chicago, United StatesAlso Flower Mound, NorfolkAge 0 to 0 - Salanersen for babies with spinal muscular atrophy after gene therapy
Tests salanersen in babies with spinal muscular atrophy already treated with onasemnogene abeparvovec.
Flower Mound, United StatesAge 0 to 0 - ARGX-119 given by infusion to children with spinal muscular atrophy
Tests ARGX-119 given through a vein in children with spinal muscular atrophy.
Baltimore, United StatesAlso Boston, Chicago, Columbia, Flower Mound, Hartford, Hillsborough, Houston, Iowa City, …Age 5 to 17 - Testing apitegromab in babies with spinal muscular atrophy
Tests apitegromab in babies under 2 with spinal muscular atrophy.
Atlanta, United StatesAlso Aurora, Dallas, Flower Mound, Fort Worth, Grand Rapids, Houston, Iowa City, Orange, …Up to age 2 - Salanersen Study in Teens and Adults With Spinal Muscular Atrophy
Tests salanersen's effect on movement in teens and adults with spinal muscular atrophy.
Norfolk, United StatesAlso Palo AltoAge 15 to 60 - Studies how the body processes nusinersen given through an implanted port
Tests nusinersen given through an implanted port in children and adults with SMA.
Boston, United StatesAlso Charlottesville, Chicago, Grand Rapids, Hershey, Houston, Norfolk, Orange, Orlando, …Age 3+ - Studies health of pregnant people with spinal muscular atrophy who took nusinersen
Observes pregnancies and babies in people with SMA who took nusinersen before or during pregnancy.
Boston, United StatesAlso Cambridge, New York, Palo AltoAge 18 to 54 - Intensive Movement and Strength Training Camp for Children With Spinal Muscular Atrophy
Tests an intensive movement and strength training camp in children with spinal muscular atrophy.
New York, United StatesAge 5 to 17 - Registry of Spinal Muscular Atrophy Diagnosed Before Birth and Treated With Medication
Follows pregnancies and babies with spinal muscular atrophy treated before birth.
Memphis, United States - Measuring Tiredness From Activity in People With Spinal Muscular Atrophy
Studies a new questionnaire on fatigue in people with spinal muscular atrophy treated with nusinersen.
New York, United StatesAge 12+ - Studies how spinal muscular atrophy drugs affect muscle in patients
Looks at how spinal muscular atrophy treatments affect muscles and nerve cells in patients.
Memphis, United StatesAge 5 to 20 - Tracking walking with digital tools in two childhood muscle diseases
Studies walking measured by digital tools in children with Duchenne or spinal muscular atrophy.
Boston, United StatesAlso New York, Palo AltoAge 5+ - Studying walking patterns and bone health in spinal muscular atrophy
Looks at how walking patterns relate to bone health in people with spinal muscular atrophy.
New York, United StatesAge 8 to 50 - Long-term registry following people with spinal muscular atrophy and their treatment outcomes
Follows people with spinal muscular atrophy over time, including those given Zolgensma.
Akron, United StatesAlso Aurora, Austin, Charlottesville, Cincinnati, Cleveland, Columbia, Columbus, Dallas, …
Where treatment stands
There is no cure yet, but there are medicines that target the changed genes that cause SMA, and other treatments help manage the symptoms.
Approved: 4
Officially approved for this disease in the EU, the US, Switzerland or France.
Human testing stage: 2
Being tested in people. Many treatments at this stage are never approved.
Animal testing stage: 5
Worked in animals in a study published in the last 12 months. Most treatments at this stage never reach people.
Placebo: a dummy treatment without the medicine, used for comparison.
Phase 1, 2, 3: the steps of testing in people. Phase 1 checks safety, phase 3 is the last step before approval.
The EU medicines agency (EMA) and its list of medicines in the EU, US prescribing labels (DailyMed, openFDA), the Swiss medicines register (Swissmedic), the French medicines database (ANSM) and the Health Canada drug database, checked on 11 November 2026. Every medicine links to its own official text.
A computer made this list from the official texts: it kept only medicines whose own approval text names the disease, wrote "Who it is for" from that text and checked it against the text a second time. People have not reviewed it yet.
The trials: ClinicalTrials.gov and the EU trials register, as copied into our trial database, checked on 12 November 2026. Every treatment in trials links to its own trial records; the texts are written by AI from them and checked against them by a second AI. People have not reviewed them yet. Placebo, standard medicines used for comparison and procedures are left out.
Phase 3 trials are listed from every country; only some phase 2 trials are listed, so 3 other open phase 2 trials are not listed.
Animal and lab research: studies listed in PubMed and published in the 12 months before 12 November 2026, found by a search for spinal muscular atrophy. AI chose the treatments from the study summaries and wrote the texts; a second AI checked each one against its study summary. People have not reviewed them yet. Treatments already in the approved or human testing lists are left out. A result in animals or cells does not show that a treatment works in people.
Not medical advice. Talk to your doctor before you change any treatment.
The approval records under a medicine come from Drugs@FDA and the FDA labels (openFDA) and from the EU medicines agency’s medicine data, with its orphan designations, checked on 13 Nov 2026. A date is the medicine’s first approval, for any use; the quoted text is its approved use as written today.
Support organisations
Patient organisations and helplines in United States, for you and your family.
Spinal muscular atrophy organisations in United States
- Cure SMA
US nonprofit supporting patients and families affected by spinal muscular atrophy and funding SMA research.
- Genetic and Rare Diseases Information Center
NIH information center (GARD) providing reliable information on genetic and rare diseases to patients and families via website and contact center.
- Muscular Dystrophy Association
US nonprofit advancing research, care and advocacy for people living with muscular dystrophy, ALS and related neuromuscular diseases.
General support in United States
- 988 Suicide & Crisis Lifeline
US national 24/7 free and confidential lifeline for anyone in suicidal crisis or emotional distress, reachable by calling or texting 988 or by chat.
- MedlinePlus
Free, easy-to-read health information service of the US National Library of Medicine (NIH) on diseases, conditions, drugs and wellness.
International organisations
Offer tissue or data
There are three ways to help research move faster.
Treatments approved outside the EU
Medicines for spinal muscular atrophy approved in the US or Japan that are not approved in the EU.
Checked on 11 Nov 2026 in the official lists of the US (FDA), Japan (PMDA, approvals since April 2004) and the EU (EMA).
Your health insurer may pay part of the cost, sometimes all of it. This depends on your insurer, your plan and the country, so always ask your insurer before you go. For planned care in the EU, the EEA and Switzerland, ask about form S2.
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“Cures” others already fact-checked
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About spinal muscular atrophy
Sources: NHS, MedlinePlus Genetics
An inherited condition in which the nerve cells that control movement are lost, so the muscles become weak and waste away. There is no cure yet, but medicines that target the altered genes, and other care, can help. Symptoms usually start in babies and toddlers, but can start in teenagers or adults. It usually happens when both parents carry an altered gene but do not have the condition themselves.
What is it?
In SMA, the nerve cells that control movement (motor neurones) are lost. The muscles then become weak and waste away, mostly those near the middle of the body. Most types are caused by a change in a gene called SMN1. A second, similar gene, SMN2, makes a little of the needed protein, so people with more copies of it usually have a milder form. SMA does not affect intelligence.
It is rare: about 1 in 8,000 to 10,000 people worldwide have it. Parents usually do not have SMA but each carry one altered gene. If both parents are carriers, each pregnancy has a 1 in 4 chance of a child with SMA, a 2 in 4 chance of a child who is a carrier, and a 1 in 4 chance of a child who neither has nor carries it.
The first signs
Symptoms usually start in babies and toddlers, and sometimes in teenagers or adults. SMA is grouped into types by when it starts: type 1 before 6 months, type 2 at 6 to 17 months, type 3 at 18 months to 17 years, and type 4 in adults.
- weak or floppy arms and legs;
- trouble sitting up, crawling or walking;
- problems breathing or swallowing;
- twitching or shaking muscles;
- bone and joint problems, such as a curved spine.
How doctors find out
A blood test can be used to confirm SMA. Babies may be offered a check as part of the newborn blood spot test. If you are at risk of having a child with SMA, a family doctor may refer you to a genetic counsellor to discuss the risks and options. Tests are available before, during and after pregnancy.
Current treatment
Nusinersen, risdiplam or onasemnogene abeparvovec target the altered genes where suitable for age and symptoms, alongside physiotherapy and help with breathing, feeding and the spine.
Will it get worse?
Yes, SMA gets worse over time, and it is usually more severe the earlier it starts. How it goes for each person depends on the symptoms and on how well treatment works. MedlinePlus Genetics describes the types like this: most children with type 1 cannot sit without help, and most do not live beyond early childhood; children with type 2 can sit but cannot stand or walk without help, and many live into their twenties or thirties; people with type 3 can walk at first but many later need a wheelchair, and people with types 3 and 4 usually have a normal life expectancy.
Medicines: what helps, and what to watch for
Medicines that target the altered genes include nusinersen (Spinraza), risdiplam (Evrysdi) and onasemnogene abeparvovec (Zolgensma). Whether one is suitable depends on age and symptoms, and it is not always possible to use them.
Good to know:
- your care team will talk with you about the possible risks and benefits of each medicine;
- people with SMA are usually advised to have the flu and pneumococcal vaccines;
- your care team makes a care plan that fits your needs.
Exercise and therapy help too
Support is available to help people with SMA have the best possible quality of life:
- physiotherapy and exercises keep muscles as strong as possible and stop joints getting stiff;
- walking frames, wheelchairs, splints, braces or shoe inserts help with moving;
- breathing exercises, machines that clear mucus, or air given through a mask help with breathing;
- changes to food, or a feeding tube through the nose or into the stomach, help with swallowing.
Spine surgery, for some people
SMA can cause a curved spine. Support for spine problems may include a back brace or surgery.
Is there a cure?
Treatments and support aim to improve quality of life, and research into new treatments is going on.
Median survival
No reliable published figure was found.
Sources
Written in plain words by Frontlight from these pages: