Stem Cell Therapy for Muscular Dystrophy: What It Involves, Who It Is For, and What the Results Show

Are You Considering Stem Cell Treatment for Muscular Dystrophy?

Muscular dystrophies are inherited muscle disorders that have limited curative treatment options, with most only focusing on delaying disease progression and preventing further muscle damage or risk of respiratory infections in order to ensure a better quality-of-life for such individuals. Stem cell treatment is being studied as a way to add to that, with studies reporting that cell therapy can improve or preserve different muscular functions which are impaired in different forms of muscular dystrophies (1, 2, 3).

Read on to see if Muscular Dystrophy Stem Cell Treatment might be right for you.

Is Stem Cell Therapy a Cure for Muscular Dystrophy?

No. Stem cell therapy does not cure muscular dystrophy.

Muscular dystrophies are caused by faults in genes needed for healthy muscle, and the umbilical cord cells we use do not correct those genes. What the published studies and our own follow-up data describe is improvement or stabilisation of specific functions, such as strength, balance and range of movement, in many patients, though not in all. The disease continues to progress. Clinical benefit is not guaranteed, the degree of improvement differs considerably from one patient to another, and the treatment remains experimental (1, 2, 3).

What the most recent research shows

For the type of cells we use, the published evidence is at an early stage and is mainly about safety. A 2025 phase 1 study from Korea gave six boys with Duchenne a single intravenous dose of donor umbilical cord (Wharton’s jelly) mesenchymal stem cells. All 18 adverse events were mild and none was serious, so the treatment was well tolerated. Over 12 weeks it showed no sign of benefit: motor scores and walking distance declined, and creatine kinase dipped and then returned to baseline. It was a single dose with no control group, and the same group has registered a larger randomised trial with repeated dosing (report summary, 12).

The reason mesenchymal stem cells are studied in muscular dystrophy comes from laboratory work. In a 2023 mouse study from Japan, weekly intravenous doses of amnion-derived mesenchymal stromal cells given to mdx mice, the standard mouse model of Duchenne, lowered inflammation in the muscle and preserved grip strength and running distance for up to a year compared with untreated mice. The cells had disappeared from the muscle within ten weeks, muscle scarring was not reduced, and no dystrophin was restored (report summary, 14). Results in mice do not transfer directly to patients, and the cells in that study were not umbilical cord cells, but the work supports the idea that these cells act by calming inflammation rather than by turning into muscle.

The strongest evidence that any cell therapy can slow Duchenne comes from a different product. HOPE-3, a phase 3 double-blind, placebo-controlled trial published in The Lancet in 2026, gave 106 boys and young men with advanced Duchenne muscular dystrophy repeated intravenous infusions of deramiocel, a heart-derived cell product, or placebo. Decline in arm function was about 54% slower with the cells (p = 0.029). The main heart endpoint was not met and the primary analysis was changed before unblinding. Side effects were mostly mild to moderate: headache, cough, fever, nausea and a fast heartbeat were the most common, and allergic-type reactions occurred in 41.5% of treated patients against 15.4% on placebo, despite pre-treatment with steroids and antihistamines (report summary, 15). The earlier HOPE-2 phase 2 trial (20 patients) had pointed the same way, and one boy in it had a severe allergic reaction that needed adrenaline (report summary, 16). Deramiocel is not a mesenchymal stem cell product and is not what we use. Its results show that one cell therapy can slow Duchenne, and they cannot be assumed to apply to other cell products.

A 2025 pilot study of DT-DEC01, a laboratory-fused muscle cell product, reported gains in three non-ambulatory patients that peaked and then faded, in a company-funded study with no control group (report summary). A 2025 critical review of the whole field is sceptical of mesenchymal stem cells for Duchenne, points out that the early myoblast transfer trials were negative overall, and argues that the main challenge is getting enough cells to engraft in muscle (report summary).

At present, one cell product has phase 3 evidence of slower decline. For umbilical cord mesenchymal stem cells the safety data are good and the mechanism is supported by animal work, but there is no controlled evidence of benefit in patients yet. All of our summaries are listed on the muscular dystrophy medical reports index.

Who Is a Candidate for Stem Cell Treatment for Muscular Dystrophy?

• Timing: There is no specific timing for stem cell treatment, but we generally recommend seeking it early after diagnosis, during childhood. The earlier the intervention, the easier it is to limit further damage to the muscle fibres before permanent wasting takes place.
• Different types of muscular dystrophy: We see patients with Duchenne, Becker and other forms. The published cell therapy trials are almost all in Duchenne, so for other types there is less evidence to guide expectations (3, 6).
• Heart and breathing: Cardiac and respiratory function are part of the medical review, because they determine whether travel and treatment are safe (2, 4, 5).
• Standard care: Steroids, physiotherapy and any approved gene-targeted treatment continue as prescribed. Stem cell therapy is not a replacement for them (3, 4, 5).

Suitability is decided case by case. Every enquiry goes to our medical department, which reviews the patient’s medical condition through our online medical evaluation system and only then recommends a specific treatment location and protocol. We recommend speaking to our specialists before you decide, so that you know what can realistically be expected in your case.

Send us the medical records and our team will come back to you.

Possible Improvements after Stem Cell Therapy for Muscular Dystrophy

How Stem Cell Therapy Improves Symptoms of Muscular Dystrophy

Stem cells are immature cells with the ability to self-renew and, depending on the type of stem cell, develop into one or more specialised cell types. Different types of stem and progenitor cells have been investigated for muscular dystrophy, including muscle-derived stem cells, mesenchymal stromal cells, bone-marrow-derived cells, and pluripotent stem-cell-derived muscle progenitors (3, 6).

One major goal of cell therapy in muscular dystrophy is to support the repair or replacement of damaged muscle fibres. Certain myogenic stem and progenitor cells can contribute directly to the formation of new muscle fibres or fuse with existing muscle fibres. Other cell types, including mesenchymal stromal cells, are thought to act mainly through the release of growth factors and other signalling molecules that support the surrounding muscle environment (3, 6).

In addition to a possible contribution to muscle repair, different stem cell approaches have been investigated for several other potential effects (3, 6):

• Supporting muscle-fibre repair and regeneration: Some myogenic stem and progenitor cells can contribute directly to new muscle formation, while other cell types may support the body’s own muscle-repair mechanisms.
• Modulating the immune system and the ongoing inflammatory process: Stem cells produce different antioxidants; thereby reducing the muscle-destructive and atrophic process characterizing different muscular dystrophies (6).
• Inhibiting the rate of fibrosis: Studies suggest that in muscular dystrophy, stem cells reduce the deposition of fibrous tissues within the muscles; therefore decreasing scar formation and the degree of muscular dystrophy. This function is reported to be due to the immune modulation capacity of stem cells – as previously mentioned (6).
• Promoting vascular supply to the muscles: by stimulating the generation of new blood vessels (neovascularization/angiogenesis) through stimulating different vascular stimulating growth factors (ex. VEGF). Better blood supply allows for better and faster muscle repair and regeneration (6).
• Preventing cell death: Through releasing substances that can inhibit the process of apoptosis, or programmed cell death, of the damaged cells until they are adequately repaired by stem cells (6, 7).

Benefits of Stem Cell Therapy in Muscular Dystrophy

Multiple studies using stem cells have reported promising results when used on such patients (8, 9, 10, 11); with one study reporting a previously wheel-chair-bound patient starting to walk supported following stem cell transplantation (9).

There has been wide variability in the source of the stem cells used as well as the route of administration for stem cells in muscular dystrophies, with positive outcomes seen across these multiple methods of administration.

Stem cell therapy in people with muscular dystrophy has resulted in (8, 9, 10, 11):

• Improved muscle strength
• Improved respiratory function
• Improved gait and mobility
• Improved balance
• Improved fine motor capabilities (ex. handwriting)
• Lower creatine kinase levels
• Enhanced number of normally-functioning muscle fibers

Safety, Side Effects and Cell Quality Control

Like any medical treatment, stem cell therapy can have side effects. Early clinical studies suggest that mesenchymal stem cell treatment has generally been well tolerated in patients with muscular dystrophy, although the available studies are small and long-term safety data remain limited. In a 2021 Polish study of 22 patients, mostly adults with limb-girdle and other forms of muscular dystrophy, who received repeated intravenous or intrathecal infusions of umbilical cord mesenchymal stem cells, only one patient developed transient headache and lower-back pain, and one gained weight (report summary, 11). In the 2025 phase 1 study of six children with Duchenne, no treatment-related serious adverse events or dose-limiting toxicity were observed. The mild events included temporary redness and swelling at the infusion site, headache, and one boy bothered by the smell of the product (report summary, 12).

Our protocol delivers cells intravenously and by intramuscular injection into the affected muscles. Intramuscular injections can cause temporary soreness, tenderness or discomfort at the injection sites.

Larger analyses of mesenchymal stem cell infusions in other diseases point the same way. A 2020 systematic review of 55 randomised trials, with 2,696 adult patients, found no increase in death, cancer, infection or blood-clot events with the cells, although fever occurred more often (13).

Every batch of cells is screened, tested twice and traceable by code before it is released for use. The full process is described on our standards and certifications page.

Where Treatment Happens, and Under Which Regulations

Treatment is delivered at affiliated hospitals in Bangkok, Thailand and in Dongguan, China. It is not approved by the US Food and Drug Administration and is not offered in the United States. It is given under Thai and Chinese regulations for cell therapy, and the cells are prepared in laboratories with CNAS, GMP and ISO accreditation. The accreditation details and the certificates are on our standards and certifications page.

Our Treatment Program in Details

Beike is unlike any other stem cell treatment provider in the world, the reason? Since 2005, we have been developing and optimizing our stem cell treatment protocols with the concept that only a very comprehensive solution can allow our patients to truly benefit from stem cells.

We believe that stimulation through various therapies is necessary to enhance stem cell regenerative response, therefore our protocols include daily therapies to support the stem cells. Finally, we provide a wide variety and large quantities of stem cells in order to adapt to each patient specific condition and deliver maximized regenerative potential.

Our stem cell therapy for muscular dystrophy program consist in 4 to 8 simple and minimally invasive injections of umbilical cord derived stem cells. The stem cells are transplanted using two separate methods: by intravenous way using a standard IV drip system, and through intramuscular injection in the affected muscles. These two delivery methods allow for increased efficacy while ensuring safety and minimum inconvenience for the patient.

Stem Cell Treatment for Muscular Dystrophy | Stem Cell Treatment Testimonial

Rafael was diagnosed with muscular dystrophy as a child. Through stem cell treatment, he has seen a slowed disease progression. In this video he is undergoing his third comprehensive stem cell treatment procedure at our partner treatment hospital. His daily routines such as holding a glass, eating by himself, kicking a ball, typing on his phone or computer have gotten easier, leading to a better quality of life.

Watch more videos

Frequently Asked Questions

Q.Can stem cell therapy cure muscular dystrophy?

A.

No. Stem cell therapy is not a cure for muscular dystrophy. The cells we use do not correct the gene fault behind the disease, which continues to progress. What studies and our own follow-up data describe is improvement or stabilisation of specific functions for many, but not all, patients. Improvement cannot be guaranteed and differs considerably from one patient to another.

Q.Is stem cell therapy for muscular dystrophy approved by the FDA?

A.

No. Stem cell therapy for muscular dystrophy is not approved by the US Food and Drug Administration, and we do not offer it in the United States. Treatment is delivered at affiliated hospitals in Thailand and China under those countries’ regulations for cell therapy. The laboratories that prepare the cells hold CNAS, ISO and GMP accreditation, which is not the same thing as FDA approval of the treatment. More detail is on our standards and certifications page.

Q.What is Muscular Dystrophy?

A.

Muscular dystrophies are a wide group of inherited disorders characterized by gradual weakness and wasting, or atrophy, of different body muscles. There are many types of muscle dystrophy, yet the most widely known type – and unfortunately the most severe – is Duchenne muscular dystrophy (DMD) (1, 2).

Q.Symptoms of Muscular Dystrophy

A.

Symptoms of muscular dystrophy vary greatly depending on the type of defect – and therefore type of Muscular Dystrophy. For example, symptoms seen with DMD are more severe and rapidly progressing than those seen in milder forms such as Becker muscular dystrophy. The most common type of muscular dystrophy is DMD and it typically presents at around 4-5 years with (2, 4, 5):

• Gradual muscle weakness and wasting
• Delay of reaching motor milestones
• Loss of previous ambulation (ex. Standing/walking)
• Recurrent respiratory complications (due to abnormal breathing muscles)
• Cardiac abnormalities (usually by the age of 20)

As DMD progresses, the ability to walk is lost, and the most serious complications come from the heart and the breathing muscles rather than from the limb muscles themselves (2, 5).

Q.Current Treatments for Muscular Dystrophy

A.

Given that muscular dystrophies occur due to inherited genetic defects in the genes responsible for proper muscle development and functioning, its treatment options are limited, similar to lots of other inherited disorders. Current treatment options for muscular dystrophies include:

• Physiotherapy: Physiotherapy is still the main form of therapy for muscular dystrophy – along with steroids (2, 4, 5).
• Steroids: Steroids are strong anti-inflammatory drugs that can also modulate the body’s immune response. There is current evidence that early use of glucocorticoids/steroids delays the onset of wheelchair dependence. However, to this day and despite extensive studies on the topic, there are still uncertainties regarding which steroid is the best to use and the possible risk of under or over-treatment. This is also in addition to the potential side effects of prolonged steroid use such as weight gain and higher risk of infections (2, 4, 5).
• Gene Therapy: this is yet again another promising mode of therapy – similar to stem cell therapy – with multiple drugs being approved by the FDA for muscular dystrophies due to the limited amount of options available. These drugs target the gene abnormality responsible for muscular destruction and dystrophy; however given that these medications target a certain genetic mutation, they depend on a certain genetic mutation/aberration being present in the patient in order to be able to use it. For example, one drug approved known as Eteplirsen has been reported to be efficacious only in around 15% of patients with DMD who carry a certain mutation in Exon 51 of the DMD gene. This means that in children who have a gene abnormality elsewhere or even a different abnormality within the same gene – which is common – don’t benefit from this medication. Therefore, gene therapy requires a meticulous genetic assessment before its use (3).

Q.Which Stem Cells are the Best to treat Muscular Dystrophy?

A.

To date, there has not been a single study – to our knowledge – that has compared different types of stem cells, concerning safety and efficacy, particularly in patients with muscular dystrophies. However, we can summarize different stem cell sources that have been tested in these disorders. Each form of stem cells has its own benefits and drawbacks as will be mentioned. Different stem cell sources that have been tested in different muscle dystrophies – either on animals or in humans – include (3, 6):

• Mesenchymal Stem Cells: These are stem cells obtained from adipose tissues, bone marrow, or umbilical cord tissues – which we actually use at Beike. These cells are the most widely used stem cells since they can be easily produced in larger numbers to accommodate a higher number of patients and allow better efficacy, have a better response in neurological and muscular diseases, have better differentiation capacity into numerous cell types, and have better anti-cell death effect in case of degenerative conditions like muscular dystrophies that cause progressive muscle wasting – compared to other stem cells. Mesenchymal stem cells can be obtained from different sources including the umbilical cord, bone marrow, and adipose tissue.
• Muscle/Satellite Stem Cells: These are another type that has particular benefits in differentiating into the defective muscle fiber cells, of the same origin; though their use is limited in patients due to their limited migration abilities to the site of defect following their transplantation. These stem cells also require long-term suppression of immunity using immunosuppressants to prevent transplant rejection.
• Adult Pluripotent Stem Cells: These are another source that can be produced in large numbers, yet their differentiation abilities are limited.
• Embryonic Stem Cells: Another type of stem cell includes embryonic stem cells. These cells can differentiate into any cell type of the body, yet they are difficult to obtain and have ethical concerns regarding their sources.

After carefully reviewing all of the benefits and risks of each type, we have decided to use mesenchymal umbilical cord-based stem cells as our main source since they have been the most extensively studied type in neuromuscular conditions, including muscular dystrophy, with the least reported side effects (10, 11, 12). This is in addition to also using umbilical cord blood cells (hematopoietic cells) concurrently to provide additional benefits from using two types of stem cells; with each contributing to muscle and nerve regeneration through different complementary mechanisms.

In addition to the source of stem cells, there are also multiple routes of stem cell administration. Most clinical trials testing stem cell therapy in Muscular Dystrophy use either one – or both – of preferably two routes; which are (6, 8, 10, 11, 12):

• Intravenous (Into the blood)
• Intramuscular (Locally into the muscles)

Some early clinical studies have used combined systemic and local administration in an attempt to provide both widespread cell distribution and targeted delivery to affected muscles (10). At Beike, we use combined intravenous and intramuscular routes concomitantly to achieve the best results with the least possible side effects.

Q.Which Stem Cells Do We Use for Treating Muscular Dystrophy?

A.

At Beike, we use umbilical cord stem cells for muscle dystrophies, both umbilical cord-related mesenchymal/tissue and blood/hematopoietic cell samples donated from healthy mothers after a normal birth. As previously mentioned, this concomitant administration of both types of stem cells, mesenchymal and hematopoietic stem cells, provides better results.

Q.Optimum Timing for Stem Cell Therapy for Muscular Dystrophy

A.

There is no specific timing for stem cell treatment, but like many other neurological conditions, we generally recommend seeking stem cell therapy early after diagnosis – i.e. during childhood. This is because the earlier the stem cell intervention, the easier it is to prevent further damage to the muscle fibers and to be able to restore normal mobile functioning before permanent damage and muscle atrophy/wasting take place.

We still need to report that clinical benefit is not 100% guaranteed as is the case with any intervention, and consulting our specialists prior to undergoing the procedure is of utmost importance in order to gain more insight into the procedure and the estimated possibility of treatment success for your individual case.

Q.Possible Side Effects of Stem Cell Therapy for Muscular Dystrophy

A.

Of course, no treatment is without complications, and stem cell therapy is the same. However, despite its novelty, mesenchymal stem cell therapy has limited side effects, if used properly, with comparable general side effects to those experienced with regular blood transfusion or foreign organ transplantation (ex. allergic reactions, cell rejection, or fever). Additionally, in studies specifically studying stem cell therapy in patients with Muscular Dystrophy, no significant side effects were reported and none were life-threatening or had life-long consequences when mesenchymal stem cells were used (11, 12). They were also easily managed medically at the time of their occurrence.

Medically reviewed by

Dr. Mohammad Alzogool

Medical Director

Beike's Medical Director, Dr. Mohammad Alzogool, is a physician-researcher with a background in ophthalmology and regenerative medicine. His research contributions span ophthalmology, regenerative medicine, artificial intelligence, medical imaging, and evidence-based clinical guidelines. He brings a strong scientific and clinical perspective to the evaluation of emerging regenerative therapies.

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Published March 23, 2017 Last updated September 30, 2026

Published research

Research on muscular dystrophy

Plain-language summaries of independent, peer-reviewed studies. Each one links to the original publication.

• Immunomodulatory Amnion-Derived Mesenchymal Stromal Cells Preserve Muscle Function in a Mouse Model of Duchenne Muscular Dystrophy 2023
• The Use of Umbilical Cord-Derived Mesenchymal Stem Cells in Patients With Muscular Dystrophies: Results From Compassionate Use in Real-Life Settings 2021
• Deramiocel Heart-Derived Cellular Therapy in Advanced Duchenne Muscular Dystrophy (HOPE-3): A Phase 3, Randomised, Double-Blind, Placebo-Controlled Trial 2026
• Long-Term Safety and Efficacy of Systemic DT-DEC01 Cell Therapy in Non-Ambulatory Duchenne Muscular Dystrophy Patients: A 24-Month Clinical Evaluation 2025
• Cell Therapy for Duchenne Muscular Dystrophy: Promises, Challenges, and Controversies 2025
• Safety and Tolerability of Wharton's Jelly-Derived Mesenchymal Stem Cells for Patients With Duchenne Muscular Dystrophy: A Phase 1 Clinical Study 2025
• Repeated Intravenous Cardiosphere-Derived Cell Therapy in Late-Stage Duchenne Muscular Dystrophy (HOPE-2): A Multicentre, Randomised, Double-Blind, Placebo-Controlled, Phase 2 Trial 2022
• Stem cell therapy for muscular dystrophies 2020

All 16 studies on muscular dystrophy

Medical References

1. 1. Mercuri E, Bönnemann CG, Muntoni F. Muscular dystrophies. Lancet. 2019;394(10213):2025-2038. https://doi.org/10.1016/S0140-6736(19)32910-1
2. 2. Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nature Reviews Disease Primers. 2021;7:13. https://doi.org/10.1038/s41572-021-00248-3
3. 3. Roberts TC, Wood MJA, Davies KE. Therapeutic approaches for Duchenne muscular dystrophy. Nature Reviews Drug Discovery. 2023;22(11):917-934. https://doi.org/10.1038/s41573-023-00775-6
4. 4. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurology. 2018;17(3):251-267. https://doi.org/10.1016/S1474-4422(18)30024-3
5. 5. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurology. 2018;17(4):347-361. https://doi.org/10.1016/S1474-4422(18)30025-5
6. 6. Biressi S, Filareto A, Rando TA. Stem cell therapy for muscular dystrophies. Journal of Clinical Investigation. 2020;130(11):5652-5664. https://doi.org/10.1172/JCI142031
7. 7. Kwon SJ, Ki SM, Park SE, et al. Anti-apoptotic effects of human Wharton's jelly-derived mesenchymal stem cells on skeletal muscle cells mediated via secretion of XCL1. Molecular Therapy. 2016;24(9):1550-1560. https://doi.org/10.1038/mt.2016.125
8. 8. Sharma A, Sane H, Badhe P, Gokulchandran N, Kulkarni P, Lohiya M, et al. A clinical study shows safety and efficacy of autologous bone marrow mononuclear cell therapy to improve quality of life in muscular dystrophy patients. Cell Transplantation. 2013;22(Suppl 1):127-138. https://doi.org/10.3727/096368913X672136
9. 9. Sharma A, Sane H, Paranjape A, Bhagawanani K, Gokulchandran N, Badhe P. Autologous bone marrow mononuclear cell transplantation in Duchenne muscular dystrophy – a case report. American Journal of Case Reports. 2014;15:128-134. https://doi.org/10.12659/AJCR.890078
10. 10. Dai A, Baspinar O, Yeşilyurt A, et al. Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy – Phase I-II. Degenerative Neurological and Neuromuscular Disease. 2018;8:63-77. https://doi.org/10.2147/DNND.S170087
11. 11. Świątkowska-Flis B, Zdolińska-Malinowska I, Sługocka D, Boruczkowski D. The use of umbilical cord-derived mesenchymal stem cells in patients with muscular dystrophies: Results from compassionate use in real-life settings. Stem Cells Translational Medicine. 2021;10(10):1372-1383. https://doi.org/10.1002/sctm.21-0027
12. 12. Lee J, Park SE, Kim M, et al. Safety and tolerability of Wharton's jelly-derived mesenchymal stem cells for patients with Duchenne muscular dystrophy: A phase 1 clinical study. Journal of Clinical Neurology. 2025;21(1):40-52. https://doi.org/10.3988/jcn.2024.0299
13. 13. Thompson M, Mei SHJ, Wolfe D, et al. Cell therapy with intravascular administration of mesenchymal stromal cells continues to appear safe: An updated systematic review and meta-analysis. EClinicalMedicine. 2020;19:100249. https://doi.org/10.1016/j.eclinm.2019.100249
14. 14. Nitahara-Kasahara Y, Nakayama S, Kimura K, et al. Immunomodulatory amnion-derived mesenchymal stromal cells preserve muscle function in a mouse model of Duchenne muscular dystrophy. Stem Cell Research & Therapy. 2023;14:108. https://doi.org/10.1186/s13287-023-03337-0
15. 15. McDonald CM, Villa C, Soslow JH, et al. Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. Lancet. 2026. Published online July 29, 2026. https://doi.org/10.1016/S0140-6736(26)01385-1
16. 16. McDonald CM, Marbán E, Hendrix S, et al. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022;399(10329):1049-1058. https://doi.org/10.1016/S0140-6736(22)00012-5