MOTS-C and SS-31: A Detailed Guide to Mitochondrial Peptide Research

Mitochondria are often described as the powerhouses of the cell, but this familiar definition captures only part of their biological importance.

These specialised cellular structures help convert nutrients into adenosine triphosphate, or ATP—the chemical energy used to support muscle contraction, cellular repair, nerve signalling and countless other biological processes.

Mitochondria also participate in:

• Metabolic regulation
• Cellular stress responses
• Calcium signalling
• Inflammatory pathways
• Reactive oxygen species production
• Programmed cell death
• Communication between mitochondria and the cell nucleus

This broader understanding has created a growing field of research focused on mitochondrial peptides.

Two of the most discussed compounds in this field are MOTS-C and SS-31, also known as elamipretide. Both are associated with mitochondrial biology, but they are structurally and functionally different.

MOTS-C is a naturally occurring mitochondrial-derived peptide encoded within mitochondrial DNA. SS-31 is a synthetic mitochondria-targeting peptide designed to interact with cardiolipin inside the inner mitochondrial membrane.

This guide examines how MOTS-C and SS-31 work, the quality of the available evidence, their potential research applications and the important differences between laboratory findings, human clinical trials and approved medical use.

What is MOTS-C?

MOTS-C, usually written as MOTS-c in scientific literature, is a naturally occurring mitochondrial-derived peptide consisting of 16 amino acids.

Its full name is “mitochondrial open reading frame of the 12S rRNA type-c.”

Most human proteins are encoded by genes located within the cell nucleus. MOTS-C is unusual because its genetic sequence is found within mitochondrial DNA, specifically within the region associated with the mitochondrial 12S ribosomal RNA gene.

MOTS-C is part of a wider group of compounds known as mitochondrial-derived peptides. Other members of this group include humanin and small humanin-like peptides.

These peptides have changed the traditional view of mitochondria. Rather than being passive energy-producing structures, mitochondria appear capable of generating signalling molecules that communicate with other parts of the cell.

What does MOTS-C do in the body?

Researchers believe endogenous MOTS-C acts as a metabolic signalling molecule.

Endogenous means that the peptide is naturally produced within the body. This is different from externally administered synthetic MOTS-C, for which human safety and efficacy have not yet been established.

MOTS-C research has examined possible roles in:

• Glucose metabolism
• Insulin sensitivity
• Skeletal-muscle function
• Metabolic flexibility
• Cellular stress adaptation
• Mitochondrial communication
• Inflammatory regulation
• Age-related metabolic changes

The peptide appears to become particularly relevant during periods of metabolic stress, when cells must adjust how they generate and use energy.

How does MOTS-C work?

The complete MOTS-C mechanism is still being investigated. Current evidence suggests that its activity involves cellular energy sensing, one-carbon metabolism and communication between mitochondria and the nucleus.

MOTS-C and AMPK activation

One of the pathways most frequently associated with MOTS-C is AMP-activated protein kinase, or AMPK.

AMPK functions as a cellular energy sensor. It becomes more active when energy availability is low and the ratio of AMP or ADP to ATP rises.

Once activated, AMPK can help the cell restore its energy balance by:

• Increasing glucose uptake
• Supporting fatty-acid oxidation
• Promoting mitochondrial adaptation
• Reducing energy-intensive biosynthetic processes
• Influencing autophagy and cellular maintenance
• Regulating metabolic gene expression

Preclinical studies suggest MOTS-C can influence AMPK partly through changes in folate and methionine metabolism, leading to an increase in the purine-intermediate AICAR.

AICAR can activate AMPK, creating a possible connection between MOTS-C, cellular energy sensing and metabolic adaptation.

This mechanism is based largely on cellular and animal research. It does not prove that externally administered MOTS-C produces a clinically meaningful AMPK effect in humans.

MOTS-C and mitochondrial-to-nuclear communication

MOTS-C may also act through mitochondrial retrograde signalling.

Most mitochondrial proteins are produced from instructions supplied by nuclear DNA. This communication from the nucleus to the mitochondria is sometimes described as anterograde signalling.

Retrograde signalling operates in the opposite direction: mitochondria send information back to the nucleus.

Research suggests that during metabolic or oxidative stress, MOTS-C can move into the cell nucleus and interact with transcription factors. It may then influence nuclear genes involved in:

• Antioxidant responses
• Protein quality control
• Glucose metabolism
• Cellular protection
• Adaptation to metabolic stress

This activity makes MOTS-C scientifically unusual. Although its sequence originates within mitochondrial DNA, some of its proposed effects involve regulating genes located within the nucleus.

MOTS-C and glucose metabolism

MOTS-C was initially identified through research examining mitochondrial regulation of metabolic homeostasis.

A foundational 2015 study found that MOTS-C influenced glucose metabolism in skeletal muscle and helped protect mice against diet-induced obesity and insulin resistance.

In these preclinical models, MOTS-C appeared to increase glucose utilisation and improve metabolic homeostasis without functioning as insulin itself.

Subsequent research has examined associations between naturally occurring MOTS-C levels and:

• Insulin resistance
• Type 2 diabetes
• Obesity
• Metabolic syndrome
• Age-related metabolic decline

Some human observational studies have reported different circulating MOTS-C levels in people with metabolic disease compared with healthy control groups. However, an association between a naturally occurring biomarker and disease does not establish that administering the biomarker will treat the disease.

Controlled human interventional trials are required to answer that question. For related reading on MOTS-C alongside the investigational triple agonist Retatrutide, see our Retatrutide and MOTS-C research.

MOTS-C and exercise research

MOTS-C is frequently described online as an “exercise mimetic.” This term should be used cautiously.

A 2021 study published in Nature Communications examined the relationship between MOTS-C and exercise.

In a small group of healthy young men, endogenous MOTS-C levels increased in skeletal muscle following exercise. Circulating levels also temporarily increased during and after exercise before returning towards baseline.

The same study found that administered MOTS-C improved physical performance, skeletal-muscle metabolism and adaptation to metabolic stress in mouse models.

These were two different findings:

1. Exercise influenced the body’s naturally occurring MOTS-C levels in humans.
2. Administered MOTS-C affected physical performance in mice.

The study did not establish that synthetic MOTS-C improves physical performance, endurance or recovery in humans.

Calling MOTS-C an exercise replacement or clinically proven performance compound would therefore go beyond the evidence.

MOTS-C and ageing research

Mitochondrial function changes as part of the ageing process. These changes may affect energy production, muscle function, oxidative balance and the ability of cells to adapt to stress.

This has made MOTS-C a subject of interest in longevity and healthy-ageing research.

Animal and cellular studies have investigated possible relationships between MOTS-C and:

• Age-related physical decline
• Muscle homeostasis
• Mitochondrial function
• Metabolic flexibility
• Oxidative stress
• Cellular senescence
• Pancreatic beta-cell health
• Inflammatory signalling

A 2025 study reported that MOTS-C levels declined with ageing and cellular senescence in mouse pancreatic islet cells. MOTS-C treatment affected gene expression and metabolites associated with beta-cell senescence in laboratory and animal models.

The study also found lower circulating MOTS-C levels in people with type 2 diabetes than in healthy controls.

These findings add to the scientific case for further investigation, but they do not demonstrate that MOTS-C prevents ageing, extends human lifespan or treats diabetes.

MOTS-C should therefore be described as an investigational mitochondrial peptide—not as a proven longevity treatment.

What is SS-31?

SS-31 is a synthetic mitochondria-targeting tetrapeptide. A tetrapeptide is a compound made from four amino-acid components.

SS-31 has been known by several names during its development:

• SS-31
• Elamipretide
• Bendavia
• MTP-131
• Forzinity, for its specific US-approved medical use

The “SS” name comes from the Szeto-Schiller family of peptides, named after researchers Hazel Szeto and Peter Schiller.

Unlike MOTS-C, SS-31 is not encoded by mitochondrial DNA and is not a naturally occurring human mitochondrial peptide. It was engineered to enter cells and concentrate around the inner mitochondrial membrane.

How does SS-31 work?

The central SS-31 mechanism involves a mitochondrial phospholipid called cardiolipin.

Cardiolipin is found predominantly within the inner mitochondrial membrane. It helps organise the membrane’s structure and supports the proteins responsible for oxidative phosphorylation.

Oxidative phosphorylation is the process through which mitochondria use the electron transport chain to generate ATP.

SS-31 is thought to bind selectively to cardiolipin through a combination of electrostatic and hydrophobic interactions.

SS-31 and cardiolipin

Cardiolipin supports several critical mitochondrial functions:

• Maintaining the structure of mitochondrial cristae
• Organising respiratory-chain complexes
• Supporting efficient electron transfer
• Helping produce ATP
• Regulating cytochrome c
• Participating in cell-death signalling

Cristae are folds within the inner mitochondrial membrane. These folds increase the membrane area available for the machinery involved in ATP production.

When cardiolipin becomes oxidised, damaged or structurally disorganised, mitochondrial energy production may become less efficient. This can also increase electron leakage and reactive oxygen species generation.

By interacting with cardiolipin, SS-31 is being studied for its potential to stabilise the inner mitochondrial membrane and preserve cristae structure.

SS-31 and cytochrome c

Cytochrome c normally carries electrons between respiratory complexes in the electron transport chain.

Under conditions of mitochondrial stress, interactions between cytochrome c and damaged cardiolipin may cause cytochrome c to adopt peroxidase-like activity. This can encourage further cardiolipin oxidation and contribute to mitochondrial dysfunction.

Research suggests SS-31 may help preserve the normal electron-carrying role of cytochrome c while reducing the cardiolipin-associated peroxidase activity linked with oxidative damage.

SS-31 and ATP production

SS-31 is not a direct source of energy, nor does it contain ATP.

Its proposed action is to improve the environment in which mitochondrial energy production occurs.

By stabilising cardiolipin and respiratory-chain organisation, SS-31 may support:

• Electron transport efficiency
• Mitochondrial membrane structure
• Oxidative phosphorylation
• ATP-generating capacity
• Coupling between respiration and ATP synthesis

These effects have been demonstrated most consistently in laboratory and animal models. Results in human studies have varied according to the condition, study design and clinical endpoint.

Is SS-31 an antioxidant?

SS-31 is often called a mitochondria-targeted antioxidant, but this description is incomplete.

Traditional antioxidants generally act by chemically neutralising reactive molecules. SS-31 appears to work further upstream by interacting with cardiolipin and influencing mitochondrial membrane structure, electron transport and reactive oxygen species generation.

Its proposed antioxidant effects may therefore arise partly from improving mitochondrial efficiency and reducing electron leakage rather than simply scavenging every reactive oxygen species directly.

SS-31 and mitochondrial oxidative stress

Reactive oxygen species are natural by-products of cellular metabolism. At controlled levels, they participate in normal signalling.

Problems can develop when their production exceeds the cell’s antioxidant and repair capacity. This imbalance is known as oxidative stress.

Excessive mitochondrial oxidative stress can affect:

• Membrane lipids
• Mitochondrial DNA
• Respiratory-chain proteins
• ATP production
• Inflammatory pathways
• Cellular survival

Preclinical SS-31 research has examined models of heart failure, kidney injury, skeletal-muscle ageing, neurodegeneration, metabolic disease and ischaemia-reperfusion injury.

Encouraging findings in these models provide a rationale for clinical research. They do not establish SS-31 as a general treatment for mitochondrial dysfunction in humans.

SS-31 and primary mitochondrial myopathy research

Primary mitochondrial myopathies are genetic disorders in which impaired mitochondrial function particularly affects skeletal muscle.

Symptoms can include:

• Muscle weakness
• Exercise intolerance
• Fatigue
• Mobility impairment
• Progressive external ophthalmoplegia
• Multisystem complications

Elamipretide was evaluated in the phase 3 MMPOWER-3 trial involving people with genetically confirmed primary mitochondrial myopathy.

The principal outcomes included distance covered during a six-minute walk test and patient-reported fatigue.

At 24 weeks, the trial did not show a statistically significant improvement in either primary endpoint compared with placebo.

This negative result is important. It demonstrates why a plausible mechanism and encouraging early research cannot be treated as proof of clinical effectiveness.

A later post-hoc analysis suggested that a genetically defined subgroup might have responded differently. Post-hoc findings can generate hypotheses, but they do not replace a successful prespecified clinical endpoint and require confirmation in appropriately designed trials.

SS-31 and heart-failure research

Mitochondrial energy deficiency has been investigated as a possible contributor to heart failure.

An early clinical study of a single intravenous infusion of elamipretide reported favourable short-term changes in left-ventricular volumes at the highest investigated exposure.

This led to further research.

In the phase 2 PROGRESS-HF trial, elamipretide was generally well tolerated but did not significantly improve the primary measure of left-ventricular end-systolic volume after four weeks compared with placebo.

These findings do not rule out all possible cardiac applications, but they show that the clinical evidence has been mixed.

SS-31 should not be described as a proven general treatment for heart failure.

SS-31, elamipretide and Barth syndrome

Barth syndrome is a rare genetic mitochondrial disorder caused by variants in the TAZ gene.

The condition primarily affects males and can involve:

• Cardiomyopathy
• Skeletal-muscle weakness
• Exercise intolerance
• Fatigue
• Growth abnormalities
• Neutropenia
• Abnormal cardiolipin metabolism

Because Barth syndrome directly affects cardiolipin remodelling, it represents a particularly relevant condition for a cardiolipin-targeting compound such as elamipretide.

The TAZPOWER trial

TAZPOWER was a small randomised, double-blind, placebo-controlled crossover trial followed by a long-term open-label extension.

The initial placebo-controlled stage did not demonstrate significant improvement in its main six-minute walk and fatigue endpoints.

During the subsequent open-label extension, participants receiving elamipretide showed sustained changes in functional assessments, muscle strength and certain cardiac measures.

Open-label extensions are valuable for studying longer-term outcomes and safety, particularly in rare diseases. However, they lack a continuing blinded placebo group and can be affected by selection, expectation, natural-history and survivorship biases.

FDA approval of elamipretide

In September 2025, the US Food and Drug Administration granted accelerated approval to Forzinity (elamipretide) for Barth syndrome in patients weighing at least 30 kilograms.

The approval was based on improvement in knee-extensor muscle strength, considered reasonably likely to predict clinical benefit.

Accelerated approval allows access based on a surrogate or intermediate endpoint considered likely to predict benefit. It normally requires additional confirmatory research.

The FDA therefore requires a post-approval randomised, double-blind, placebo-controlled trial to determine whether the observed muscle-strength changes translate into meaningful patient benefits, such as improved standing or walking.

This approval applies to a defined medical product, indication and patient population. It does not mean that SS-31 has been approved for:

• General mitochondrial optimisation
• Longevity or anti-ageing
• Athletic performance
• Fat loss
• Everyday fatigue
• Heart failure
• Neurodegenerative disease
• Primary mitochondrial myopathy generally

The distinction between a regulated medicine and an online product labelled “SS-31” is critical.

SS-31 side effects and safety research

In elamipretide clinical studies, the most frequently reported adverse effects have included injection-site reactions.

These can include:

• Redness
• Pain
• Itching
• Swelling
• Irritation
• Bruising

The FDA has also stated that serious reactions have been reported.

Safety data from a pharmaceutical product manufactured under regulated conditions cannot automatically be applied to unregulated research material. Differences in identity, purity, concentration, sterility, storage and contamination could materially change the risk.

Long-term safety may also vary according to the population, medical condition, route of administration and duration of exposure.

MOTS-C vs SS-31: what is the difference?

Although both are called mitochondrial peptides, MOTS-C and SS-31 are not interchangeable.

Research area	MOTS-C	SS-31
Alternative name	MOTS-c	Elamipretide, Bendavia, MTP-131
Structure	16-amino-acid peptide	Synthetic tetrapeptide
Origin	Encoded within mitochondrial DNA	Laboratory-engineered
Primary research focus	Metabolic signalling and stress adaptation	Inner mitochondrial membrane and cardiolipin
Principal proposed pathways	AMPK, folate metabolism and nuclear gene regulation	Cardiolipin binding, cristae stability and electron transport
Human research	Mainly observational and early-stage	Multiple clinical trials
Regulatory position	Not an approved medicine	FDA-approved as Forzinity for a restricted Barth syndrome indication
General longevity approval	No	No
Established combined use	No	No

A simple way to understand the distinction is:

• MOTS-C appears to act primarily as a metabolic messenger.
• SS-31 appears to act primarily at the mitochondrial membrane.

That is a useful conceptual comparison, but both mechanisms are more complex than these short descriptions suggest.

Could MOTS-C and SS-31 be complementary?

From a mechanistic perspective, researchers may be interested in whether metabolic signalling and mitochondrial membrane stability interact.

MOTS-C is being studied for its relationship with cellular energy sensing, glucose metabolism and stress-related gene expression. SS-31 is being studied for its interaction with cardiolipin, mitochondrial structure and oxidative phosphorylation.

This theoretical distinction does not establish that the compounds are complementary when administered together.

There is currently no robust human clinical evidence demonstrating that combining MOTS-C and SS-31 is safe or effective.

Important unanswered questions include:

• Whether their biological effects interact
• Whether combined exposure alters cellular stress signalling
• Whether there are additive or opposing metabolic effects
• Whether one affects the pharmacology of the other
• Whether combined use introduces unrecognised risks
• Which biomarkers or endpoints would be appropriate
• Whether findings would vary by disease or genetic background

Until controlled research answers these questions, MOTS-C and SS-31 should be treated as separate fields of investigation rather than an established mitochondrial protocol.

MOTS-C, SS-31 and longevity claims

Both MOTS-C and SS-31 are frequently discussed within longevity communities.

The scientific rationale usually centres on the observation that mitochondrial dysfunction is associated with ageing and many age-related conditions.

However, association does not prove that a mitochondrial peptide will slow ageing.

There is currently no convincing clinical evidence that either administered MOTS-C or SS-31:

• Extends human lifespan
• Reverses biological ageing
• Prevents age-related disease in healthy people
• Replaces exercise
• Produces general “mitochondrial rejuvenation”
• Safely enhances performance in healthy adults

SS-31’s US approval for a specific rare mitochondrial disease should not be used to support unrelated anti-ageing claims.

Likewise, animal MOTS-C research involving metabolism or physical capacity should not be presented as established human longevity evidence.

The future of mitochondrial peptide research

MOTS-C and SS-31 represent two important developments in mitochondrial science.

MOTS-C has helped demonstrate that mitochondrial DNA may encode biologically active signalling peptides capable of influencing communication throughout the cell.

SS-31 has shown that mitochondrial membrane lipids such as cardiolipin can be directly targeted by carefully engineered compounds.

Future MOTS-C research needs to determine:

• Its normal physiological range in humans
• How age, sex, exercise and metabolic health affect MOTS-C
• Whether circulating levels accurately reflect tissue activity
• Its pharmacokinetics when administered externally
• Its short- and long-term human safety
• Whether preclinical metabolic findings translate to people
• Whether it produces clinically meaningful outcomes

Future SS-31 research needs to clarify:

• The benefits and risks of long-term elamipretide treatment
• The confirmatory evidence required after accelerated approval
• Which mitochondrial diseases are most likely to respond
• Whether genetic subgroups respond differently
• Why some trials have failed despite promising mechanisms
• Whether particular biomarkers can predict response
• Whether findings extend beyond Barth syndrome

The strongest scientific approach is neither to dismiss these peptides nor to overstate them. It is to distinguish clearly between mechanism, preclinical evidence, exploratory human findings, successful clinical endpoints and regulatory approval.

Frequently asked questions about MOTS-C and SS-31

What is MOTS-C peptide?

MOTS-C is a 16-amino-acid mitochondrial-derived peptide encoded within mitochondrial DNA. It is being studied for its possible roles in metabolic signalling, glucose metabolism, stress adaptation and skeletal-muscle biology.

What is SS-31 peptide?

SS-31 is a synthetic four-component peptide that targets cardiolipin within the inner mitochondrial membrane. It is also known as elamipretide.

Is SS-31 the same as elamipretide?

Yes. SS-31 was an early research name for the compound later developed as elamipretide. Bendavia and MTP-131 are additional names associated with its development.

How do MOTS-C and SS-31 differ?

MOTS-C is a naturally encoded mitochondrial signalling peptide associated with metabolic adaptation. SS-31 is a synthetic peptide designed to interact with cardiolipin and support inner mitochondrial membrane structure.

Is MOTS-C approved for human use?

MOTS-C is not an approved medicine in the UK or United States. Its proposed metabolic and longevity applications remain investigational.

Is SS-31 FDA-approved?

The FDA granted accelerated approval to elamipretide under the brand name Forzinity in September 2025. The approval is specifically for Barth syndrome in patients weighing at least 30 kilograms. It is not a general approval for mitochondrial health or longevity.

Does MOTS-C increase energy?

Laboratory and animal research suggests MOTS-C influences pathways involved in energy regulation. This does not prove that externally administered MOTS-C safely increases energy or reduces fatigue in humans.

Does SS-31 increase ATP?

Preclinical evidence suggests SS-31 may improve mitochondrial electron transport and ATP-generating efficiency under certain conditions. Clinical results have varied, and it should not be presented as a universal ATP-enhancing treatment.

Can MOTS-C improve insulin sensitivity?

MOTS-C has improved insulin sensitivity and glucose metabolism in several animal models. Controlled human evidence remains limited, so it is not an approved insulin-sensitising treatment.

Can MOTS-C and SS-31 be combined?

There is no established clinical evidence confirming the safety or effectiveness of combining MOTS-C and SS-31.

Are MOTS-C and SS-31 anti-ageing peptides?

Both are studied in areas related to mitochondrial function and age-associated biology. Neither has been proven to reverse human ageing or extend human lifespan.

NŪVO Research Perspective

MOTS-C and SS-31 illustrate how rapidly mitochondrial science is developing.

MOTS-C has expanded our understanding of mitochondria as signalling structures capable of communicating with the cell nucleus and influencing metabolic adaptation.

SS-31 has demonstrated the potential importance of cardiolipin, cristae structure and inner mitochondrial membrane organisation as research targets.

The scientific potential is substantial, but evidence must be interpreted according to its level.

Cell experiments generate mechanisms. Animal studies test biological hypotheses. Observational human studies identify associations. Randomised clinical trials evaluate clinical effects. Regulatory approval applies only to the product, population and indication that have been reviewed.

Mitochondrial energy also depends on the coenzyme NAD+, which sits at the centre of electron transfer and cellular metabolism. For a detailed companion guide, see our article on NAD+ and mitochondrial function.

Keeping these distinctions clear allows mitochondrial peptide research to be explored without turning preliminary evidence into unsupported health claims. Browse the research catalogue — every compound is supplied for laboratory research only.

Research and medical disclaimer

This article is provided for general educational and scientific-information purposes only. It does not provide medical advice, diagnosis, treatment instructions or recommendations for combining or administering compounds.

MOTS-C is not an approved medicine. Elamipretide has received accelerated FDA approval only as the regulated prescription product Forzinity for a defined Barth syndrome population in the United States.

Approval of that medical product does not establish the safety, identity or effectiveness of unregulated products labelled SS-31 or elamipretide. Readers should consult an appropriately qualified healthcare professional regarding any medical condition.

Sources and further reading

1. Lee C, et al. The mitochondrial-derived peptide MOTS-c promotes metabolic homeostasis and reduces obesity and insulin resistance. Cell Metabolism, 2015. View the study on PubMed
2. Reynolds JC, et al. MOTS-c is an exercise-induced mitochondrial-encoded regulator of age-dependent physical decline and muscle homeostasis. Nature Communications, 2021. Read the full open-access study
3. Kim KH, et al. The mitochondrial-encoded peptide MOTS-c translocates to the nucleus to regulate nuclear gene expression in response to metabolic stress. Cell Metabolism, 2018. View the study on PubMed
4. Zheng Y, Wei Z and Wang T. MOTS-c: A promising mitochondrial-derived peptide for therapeutic exploitation. Frontiers in Endocrinology, 2023. View the review on PubMed
5. Ramanjaneya M, et al. Lipids and insulin regulate mitochondrial-derived peptide MOTS-c in humans. Clinical Endocrinology, 2019. View the research on PubMed
6. Kong BS, et al. Mitochondrial-encoded peptide MOTS-c prevents pancreatic islet cell senescence. Experimental & Molecular Medicine, 2025. View the study on PubMed
7. Szeto HH. First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics. British Journal of Pharmacology, 2014. View the review on PubMed
8. Tung C, et al. Elamipretide: A review of its structure, mechanism of action and therapeutic potential. 2025. View the review on PubMed
9. Karaa A, et al. The MMPOWER-3 randomised clinical trial of elamipretide in primary mitochondrial myopathy. Neurology, 2023. View the clinical trial on PubMed
10. Butler J, et al. Elamipretide in heart failure with reduced ejection fraction: the PROGRESS-HF phase 2 trial. 2020. View the trial on PubMed
11. Vernon HJ, et al. Long-term efficacy and safety of elamipretide in patients with Barth syndrome. 2024. View the research on PubMed
12. US Food and Drug Administration. FDA grants accelerated approval to first treatment for Barth syndrome. September 2025. Read the FDA announcement