AGAMREE: Uses, Dosage & Side Effects
A dissociative anti-inflammatory steroid for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older, designed to retain muscle-protective benefits while reducing typical corticosteroid side effects
AGAMREE (vamorolone) is a novel dissociative anti-inflammatory steroid medication approved for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. Unlike traditional corticosteroids such as prednisone and deflazacort that have long been the standard of care for DMD, vamorolone was specifically designed to separate the beneficial anti-inflammatory effects of glucocorticoid receptor activation from many of the harmful side effects associated with conventional steroids. AGAMREE is taken once daily as an oral suspension and has been shown to stabilize or improve muscle strength, helping to preserve motor function in boys and young men with DMD. It also acts as a mineralocorticoid receptor antagonist, which may provide additional cardiovascular benefits relevant to DMD patients.
Quick Facts: AGAMREE
Key Takeaways
- AGAMREE (vamorolone) is a first-in-class dissociative steroid specifically developed for Duchenne muscular dystrophy, designed to provide anti-inflammatory muscle protection while reducing bone loss, growth suppression, and behavioral side effects compared to traditional corticosteroids.
- It is approved for patients aged 4 years and older with DMD and is taken once daily as an oral suspension (40 mg/mL), with the dose based on body weight – typically 6 mg/kg/day for patients under 40 kg and a fixed 240 mg/day for those 40 kg or above.
- AGAMREE causes adrenal insufficiency (reduced cortisol production), meaning it must never be stopped abruptly; doses should be tapered gradually under medical supervision to prevent potentially life-threatening adrenal crisis.
- Patients switching from another corticosteroid such as prednisone can transition to AGAMREE directly under their doctor’s guidance, without the need for a washout period between treatments.
- Live or live-attenuated vaccines (e.g., measles, mumps, rubella, varicella) must not be given during treatment or within six weeks before starting AGAMREE, as the immunosuppressive effects could trigger the very infection the vaccine is designed to prevent.
What Is AGAMREE and What Is It Used For?
AGAMREE contains the active substance vamorolone, a novel steroidal anti-inflammatory drug that belongs to a new class of medications known as dissociative glucocorticoid receptor modulators. This innovative approach to corticosteroid therapy represents a significant advancement in the treatment of Duchenne muscular dystrophy, a devastating genetic condition that causes progressive muscle weakness and wasting in boys and young men.
Duchenne muscular dystrophy is caused by mutations in the dystrophin gene, which normally produces a protein essential for maintaining the structural integrity of muscle fibers. Without functional dystrophin, muscle cells become vulnerable to damage during contraction, leading to a cycle of muscle injury, inflammation, and replacement of muscle tissue with fibrotic scar tissue and fat. This relentless process causes progressive loss of muscle strength, typically beginning in the legs and pelvis and eventually affecting the arms, respiratory muscles, and heart. Most boys with DMD lose the ability to walk by their early teens, and the condition significantly reduces life expectancy due to respiratory and cardiac complications.
Corticosteroids have been the cornerstone of DMD management for decades, with prednisone and deflazacort shown to slow muscle degeneration, prolong ambulation, preserve respiratory and cardiac function, and extend survival. However, these traditional corticosteroids come with a substantial burden of side effects, including significant weight gain, growth suppression, bone loss leading to vertebral fractures, behavioral changes such as aggression and mood swings, cushingoid appearance, cataracts, elevated blood sugar, and immunosuppression. These side effects significantly impact quality of life for patients and their families, and sometimes necessitate dose reduction or discontinuation of treatment.
Vamorolone was specifically engineered to address this therapeutic dilemma. Traditional corticosteroids exert their effects through two main mechanisms at the glucocorticoid receptor: trans-repression (suppressing inflammatory gene expression, which provides the beneficial anti-inflammatory effects) and trans-activation (activating metabolic and other genes, which drives many of the unwanted side effects). Vamorolone is designed as a “dissociative” steroid – it retains the trans-repression activity that reduces inflammation and protects muscle, while showing substantially reduced trans-activation activity. This dissociative profile means that vamorolone can provide the muscle-protective benefits of corticosteroid therapy while potentially reducing or avoiding many of the metabolic, skeletal, and behavioral side effects.
In addition to its glucocorticoid receptor modulating properties, vamorolone acts as an antagonist at the mineralocorticoid receptor. This is particularly relevant in DMD, where cardiac involvement is a major cause of morbidity and mortality. Mineralocorticoid receptor antagonists such as spironolactone and eplerenone are already used in DMD to protect the heart and cardiovascular system, so the inherent mineralocorticoid receptor antagonist activity of vamorolone may provide additional cardiovascular benefits beyond those of traditional corticosteroids.
DMD affects approximately 1 in 3,500–5,000 male births worldwide. It is an X-linked recessive genetic disorder, meaning it primarily affects boys, although in rare cases female carriers may show mild symptoms. The dystrophin gene is the largest gene in the human genome, making it particularly susceptible to mutations. Current management of DMD is multidisciplinary, involving corticosteroid therapy (now including vamorolone as an alternative to prednisone/deflazacort), cardiac monitoring and treatment, respiratory support, physiotherapy, and emerging gene and exon-skipping therapies.
What Should You Know Before Taking AGAMREE?
Contraindications
There are specific situations in which AGAMREE must not be used. Understanding these absolute contraindications is essential before starting treatment.
- Hypersensitivity: Do not take AGAMREE if you are allergic to vamorolone or any of the other ingredients in the product, including citric acid monohydrate, disodium phosphate, glycerol, orange flavoring, sodium benzoate, sucralose, xanthan gum, or hydrochloric acid.
- Severe liver disease: AGAMREE must not be used in patients with severe hepatic (liver) impairment, as the metabolism and safety of vamorolone have not been adequately studied in this population and the risk of adverse effects is increased.
- Live or live-attenuated vaccines: You must not receive live or live-attenuated vaccines (such as measles, mumps, rubella, or varicella) while taking AGAMREE or within the six weeks before starting treatment. The immunosuppressive effects of vamorolone could allow the weakened virus in these vaccines to cause the actual infection rather than providing protection against it.
Warnings and Precautions
AGAMREE suppresses your body’s natural production of cortisol (adrenal insufficiency). You must never stop taking AGAMREE suddenly or rapidly reduce the dose without consulting your doctor. Abrupt discontinuation can cause acute adrenal crisis – a potentially life-threatening condition characterized by severe fatigue, dizziness, confusion, nausea, low blood pressure, and collapse. If you need to stop AGAMREE, your doctor will gradually taper your dose over a period of time. Always carry your patient alert card, which is included in every AGAMREE package.
Before and during treatment with AGAMREE, talk to your doctor about the following:
- Stress situations: If you experience unusual physical stress such as acute infection, injury, or major surgery while taking AGAMREE, you may need supplemental steroid treatment (stress dosing) to prevent acute adrenal crisis. Discuss an emergency plan with your doctor before starting treatment so you know what to do in these situations.
- Switching from another corticosteroid: If you are currently taking another corticosteroid such as prednisone or deflazacort, you can switch to AGAMREE from one day to the next without a washout period. Your doctor will advise you on the appropriate starting dose of AGAMREE based on your previous steroid regimen.
- Weight gain: AGAMREE can cause increased appetite and weight gain, particularly during the first months of treatment. Your doctor or dietitian will provide dietary advice before and during treatment to help manage weight.
- Thyroid disorders: If you have hypothyroidism (underactive thyroid) or hyperthyroidism (overactive thyroid), your doctor may need to monitor your treatment more closely or adjust your dose, as thyroid dysfunction can affect how your body processes corticosteroids.
- Eye problems: If you or anyone in your family has glaucoma (increased pressure in the eye), your doctor may need to monitor your eye health more closely, as corticosteroids can exacerbate this condition.
- Increased infection risk: AGAMREE can reduce your natural resistance to infections. If you have an impaired immune system (due to immunodeficiency, illness, or other immunosuppressive medications), your doctor will monitor you more carefully. If you develop an infection during treatment, you may need additional steroid support and closer medical monitoring.
- Diabetes risk: Long-term use of AGAMREE may increase the likelihood of developing diabetes mellitus. Your doctor may monitor your blood sugar levels periodically throughout treatment.
- Varicella (chickenpox): If you have never had chickenpox or have not been vaccinated against it, discuss vaccination with your doctor before starting AGAMREE, as chickenpox infection can be more severe in immunosuppressed patients.
- Thromboembolic events: If you have a history of blood clots or a condition that increases your risk of developing blood clots, your doctor may need to monitor you more closely during treatment.
- Pheochromocytoma: If you have pheochromocytoma (a rare tumor of the adrenal glands), your doctor will need to manage your treatment with particular caution.
- Liver impairment: If you have mild or moderate liver disease, your doctor may need to adjust your AGAMREE dose, as the drug is metabolized primarily by the liver.
Pregnancy and Breastfeeding
Although AGAMREE is primarily used in male patients with Duchenne muscular dystrophy, the following information is important for female patients or caregivers of reproductive age. If you are pregnant, breastfeeding, think you may be pregnant, or are planning to have a baby, ask your doctor for advice before taking this medicine.
AGAMREE should not be used during pregnancy unless your doctor explicitly considers it necessary. Women of childbearing potential must use effective contraception during treatment. Animal studies have shown that long-term treatment with vamorolone may impair fertility in both males and females, which should be discussed with your healthcare provider.
Children Under 4 Years
AGAMREE should not be given to children under 4 years of age because the safety and effectiveness of vamorolone have not been studied in this age group. Your doctor will determine the appropriate time to start treatment based on your child’s age, clinical condition, and the typical trajectory of Duchenne muscular dystrophy.
Important Information About Ingredients
AGAMREE contains 1 mg of sodium benzoate (E 211) per mL. It also contains less than 23 mg of sodium per 7.5 mL dose, meaning it is essentially sodium-free. However, patients on very strict sodium-restricted diets should be aware of this small sodium content.
How Does AGAMREE Interact with Other Drugs?
Drug interactions with AGAMREE primarily involve the cytochrome P450 3A4 (CYP3A4) enzyme system, which is the main pathway for vamorolone metabolism. Medications that affect CYP3A4 activity can alter vamorolone blood levels, potentially increasing the risk of side effects or reducing therapeutic effectiveness. It is essential to tell your doctor about all medications, supplements, and herbal products you are currently taking or plan to take.
Major Interactions
| Interacting Drug | Effect | Clinical Significance |
|---|---|---|
| Itraconazole, Voriconazole (triazole antifungals) | Strong CYP3A4 inhibitors; increase vamorolone blood levels | Increased risk of side effects; avoid combination or monitor closely |
| Clarithromycin, Telithromycin (macrolide/ketolide antibiotics) | CYP3A4 inhibitors; increase vamorolone exposure | May enhance side effects; dose adjustment may be needed |
| Carbamazepine, Phenytoin (antiepileptics) | CYP3A4 inducers; decrease vamorolone blood levels | May reduce AGAMREE effectiveness; alternative treatments should be considered |
| Rifampicin (rifamycin antibiotic) | Strong CYP3A4 inducer; significantly reduces vamorolone levels | Likely to substantially reduce AGAMREE effectiveness; avoid combination |
| Live vaccines (e.g., MMR, varicella, BCG) | Risk of vaccine-strain infection due to immunosuppression | Contraindicated – do not administer during treatment or within 6 weeks before starting |
Minor Interactions
| Interacting Drug | Effect | Clinical Significance |
|---|---|---|
| Spironolactone, Eplerenone (potassium-sparing diuretics) | Overlapping mineralocorticoid receptor antagonist effects with AGAMREE | Your doctor may monitor potassium levels and adjust doses; commonly used in DMD cardiac management |
| St. John’s Wort (Hypericum perforatum) | CYP3A4 inducer; may decrease vamorolone levels | Avoid use during AGAMREE treatment; may reduce drug effectiveness |
| Grapefruit and grapefruit juice | CYP3A4 inhibitor; may increase vamorolone exposure | Avoid grapefruit products during treatment |
| Other immunosuppressive agents | Additive immunosuppression; increased infection risk | Monitor closely for signs of infection |
The interaction between AGAMREE and potassium-sparing diuretics such as spironolactone and eplerenone is particularly noteworthy in the context of DMD management. These diuretics are frequently prescribed to DMD patients for cardiac protection, and since vamorolone itself has mineralocorticoid receptor antagonist activity, there may be additive effects. Your doctor will carefully monitor your potassium levels and cardiac status if you are taking both AGAMREE and one of these medications, and may adjust the doses accordingly to optimize both cardiac protection and muscle function benefits.
What Is the Correct Dosage of AGAMREE?
AGAMREE should always be taken exactly as your doctor or pharmacist has instructed. Do not change the dose or stop taking it without first consulting your healthcare provider, as abrupt changes can lead to serious complications including adrenal crisis.
Standard Dosing
Patients Aged 4 Years and Older, Weighing Less Than 40 kg
Dose: 6 mg per kg of body weight, taken once daily
Administration: Oral suspension, measured using the provided graduated oral syringe
Example: A child weighing 25 kg would take 150 mg (3.75 mL of the 40 mg/mL suspension)
Patients Aged 4 Years and Older, Weighing 40 kg or More
Dose: 240 mg once daily (fixed dose)
Administration: Oral suspension, measured as 6 mL using the provided graduated oral syringe
How to Take AGAMREE
AGAMREE is an oral suspension (liquid) that should preferably be taken with food. Follow these steps carefully for each dose:
- Shake the bottle well before each use to ensure the medication is evenly distributed throughout the suspension.
- Use the provided oral syringe to measure your dose accurately. The syringes are graduated from 0 to 8 mL in 0.1 mL increments. Only use the syringes provided in the package – do not use any other measuring device.
- Draw up your prescribed dose into the oral syringe, then immediately and slowly empty the contents of the syringe directly into the mouth.
- After use, disassemble the syringe and plunger, rinse them under cold running tap water, and allow them to air dry. Store the clean syringe in the original packaging until the next use.
- Replace the oral syringe after 45 days of use. A second syringe is provided in each package for this purpose.
Caregivers should assist with the preparation and administration of AGAMREE, especially regarding the correct use of the oral syringes and accurate measurement of the prescribed dose. If you are unsure about any aspect of dosing or administration, ask your doctor or pharmacist for a demonstration.
Administration via Enteral Feeding Tube
AGAMREE can be administered through an enteral feeding tube (nasogastric or gastrostomy tube) following the instructions provided with the tube set. The usual prescribed dose should be used without dilution, and the medication should not be mixed with tube feeds or other products. The feeding tube must be flushed before and after administration of AGAMREE using the syringe provided with the enteral tube set, with at least 20 mL of water for each flush.
Dose Adjustments
Your doctor may adjust your AGAMREE dose in the following circumstances:
- Side effects: If you experience certain side effects, your doctor may choose to reduce the dose, temporarily interrupt treatment, or permanently discontinue AGAMREE.
- Liver impairment: If you have mild or moderate liver disease, your doctor may need to reduce the dose to account for slower metabolism of vamorolone.
- Switching from another corticosteroid: If you are transitioning from prednisone or deflazacort, your doctor will specify your AGAMREE starting dose. The switch can be made from one day to the next without a washout period.
Missed Dose
If you forget to take a dose of AGAMREE, do not take an extra dose or double the next dose to make up for it. Simply take your next dose at the usual time and continue with your regular dosing schedule. If you are concerned about a missed dose, talk to your healthcare provider for guidance.
Overdose
If you accidentally take more AGAMREE than prescribed, contact your doctor or go to a hospital emergency department immediately for advice. Bring the AGAMREE package and this information with you. Medical treatment may be necessary depending on the amount taken.
Take AGAMREE for as long as your doctor tells you to. If you need to stop treatment, your doctor must gradually reduce your dose over time to allow your adrenal glands to resume normal cortisol production. Stopping abruptly can cause acute adrenal insufficiency, which can be life-threatening. Always discuss any planned changes to your treatment regimen with your doctor before making them.
What Are the Side Effects of AGAMREE?
Like all medicines, AGAMREE can cause side effects, although not everyone gets them. Because vamorolone is a dissociative corticosteroid, its side effect profile may differ from traditional corticosteroids in important ways. Clinical studies have suggested that vamorolone may be associated with less bone loss, better growth preservation, and improved behavioral outcomes compared with prednisone, although some typical steroid-related effects still occur.
Adrenal Insufficiency
Treatment with AGAMREE leads to adrenal insufficiency, meaning your body produces less of the hormone cortisol than it normally would. This is an expected effect of the medication and is the reason why AGAMREE must never be stopped abruptly. Your doctor will provide a patient alert card with important safety information about adrenal insufficiency that you should carry with you at all times. In situations of unusual physical stress (such as illness, injury, or surgery), you may need supplemental steroid treatment to compensate for your reduced cortisol production.
Very Common
May affect more than 1 in 10 people
- Cushingoid features (a more rounded and puffy facial appearance)
- Weight gain
- Increased appetite
- Irritability
- Vomiting
Common
May affect up to 1 in 10 people
- Abdominal pain (stomach pain)
- Upper abdominal pain
- Diarrhea
- Headache
Comparison with Traditional Corticosteroids
One of the key advantages of vamorolone’s dissociative mechanism is the potential for a more favorable side effect profile compared with conventional corticosteroids like prednisone and deflazacort. Clinical studies, including the pivotal VISION-DMD trial, have shown that AGAMREE may offer the following advantages over traditional steroids:
- Bone health: Vamorolone has been associated with less bone loss compared with prednisone. Traditional corticosteroids are well known to cause osteoporosis and increase fracture risk, which is particularly concerning in DMD patients who already have reduced bone density due to limited mobility. Clinical data suggest that vamorolone may preserve bone mineral density better than conventional steroids.
- Growth: Growth suppression is one of the most distressing side effects of traditional corticosteroid therapy in children with DMD. Preliminary data suggest that vamorolone may have less impact on growth velocity compared to prednisone, although long-term studies are still ongoing.
- Behavior: Behavioral changes, including aggression, hyperactivity, and mood disturbances, are common with traditional corticosteroids and can significantly impact family life. Studies have indicated that vamorolone may be associated with fewer behavioral side effects compared to prednisone.
- Metabolic effects: While weight gain and increased appetite still occur with AGAMREE (as listed among very common side effects), the overall metabolic burden may be reduced compared to traditional corticosteroids.
However, it is important to note that AGAMREE still carries risks inherent to corticosteroid therapy, including adrenal suppression, increased infection susceptibility, and the potential for cushingoid features. Long-term post-marketing surveillance will continue to refine our understanding of the comparative safety profile.
If you experience any side effects, including those not listed here, talk to your doctor, pharmacist, or nurse. You can also report suspected side effects to your national pharmacovigilance authority (such as the EMA in Europe, the FDA MedWatch program in the United States, or the MHRA Yellow Card Scheme in the United Kingdom) to help monitor the ongoing benefit-risk profile of AGAMREE.
How Should You Store AGAMREE?
Keep AGAMREE out of the sight and reach of children at all times. Do not use this medicine after the expiry date printed on the carton and bottle label after “EXP.” The expiry date refers to the last day of the stated month.
- Before opening: No special temperature storage conditions are required. Store the bottle in the original carton to protect from light.
- After opening: Store the opened bottle upright in a refrigerator at 2°C to 8°C (36°F to 46°F). The medication can be stored under refrigeration for up to 3 months after first opening.
- Discard after 3 months: Any unused medication remaining in the bottle must be discarded within 3 months of first opening, regardless of the expiry date printed on the packaging.
- Oral syringe care: After each use, disassemble the oral syringe and plunger, rinse under cold running water, and air dry. Store the clean syringe in the original packaging. Replace the syringe after 45 days of use with the second syringe provided in the package.
Do not dispose of medications via wastewater or household waste. Ask your pharmacist how to properly dispose of medicines you no longer use. These measures help protect the environment.
What Does AGAMREE Contain?
Active Substance
The active substance is vamorolone. Each milliliter (mL) of the oral suspension contains 40 mg of vamorolone. Vamorolone is a delta-9,11 modified analogue of cortisol that has been structurally designed to function as a dissociative glucocorticoid receptor modulator and mineralocorticoid receptor antagonist.
Inactive Ingredients (Excipients)
- Citric acid monohydrate (E 330)
- Disodium phosphate (E 339)
- Glycerol (E 422)
- Orange flavoring
- Purified water
- Sodium benzoate (E 211) – 1 mg per mL (preservative)
- Sucralose (E 955) – sweetener
- Xanthan gum (E 415)
- Hydrochloric acid (for pH adjustment)
Appearance and Packaging
AGAMREE is a white to off-white oral suspension supplied in an amber glass bottle with a tamper-evident, child-resistant polypropylene closure with a low-density polyethylene liner. Each bottle contains 100 mL of oral suspension. Each package includes one bottle, a bottle adapter (to facilitate withdrawal of the medication with the syringe), and two identical oral dosing syringes graduated from 0 to 8 mL in 0.1 mL increments.
Marketing Authorization Holder
Santhera Pharmaceuticals (Deutschland) GmbH, Marie-Curie-Straße 8, D-79539 Lörrach, Germany. For further information about AGAMREE, visit the European Medicines Agency (EMA) website or consult the full Summary of Product Characteristics and prescribing information available through your regulatory authority.
Frequently Asked Questions About AGAMREE
AGAMREE (vamorolone) is used to treat Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. It is a dissociative anti-inflammatory steroid that stabilizes or improves muscle strength by reducing the inflammation and immune-mediated damage that accelerates muscle loss in DMD. Unlike traditional corticosteroids, vamorolone is designed to retain the beneficial anti-inflammatory effects while potentially reducing common steroid side effects such as bone loss, growth suppression, and behavioral changes.
AGAMREE (vamorolone) is a dissociative steroidal anti-inflammatory that was specifically designed to separate the beneficial anti-inflammatory effects of corticosteroids from many of their harmful side effects. Traditional steroids like prednisone activate both the trans-repression pathway (anti-inflammatory, beneficial) and the trans-activation pathway (responsible for many side effects). Vamorolone retains the trans-repression activity but has substantially reduced trans-activation activity. Clinical studies suggest this may result in better preservation of bone health, growth, and behavior compared with prednisone, while providing comparable muscle function benefits. Additionally, vamorolone has mineralocorticoid receptor antagonist activity, which may provide extra cardiovascular protection relevant to DMD patients.
Yes, if you are currently being treated with another corticosteroid such as prednisone or deflazacort, you can switch to AGAMREE from one day to the next without a washout period. Your doctor will determine the appropriate starting dose of AGAMREE based on your current corticosteroid dose and treatment history. It is important to follow your doctor’s instructions carefully during the transition and to report any unusual symptoms promptly.
Stopping AGAMREE suddenly can be dangerous and potentially life-threatening. Because AGAMREE suppresses your body’s natural cortisol production (adrenal insufficiency), abrupt discontinuation can trigger an adrenal crisis. Symptoms may include severe fatigue, dizziness, confusion, nausea, low blood pressure, and in severe cases, cardiovascular collapse. If treatment needs to be stopped, your doctor will gradually taper the dose over a period of time to allow your adrenal glands to slowly resume normal cortisol production. Never change your dose or stop taking AGAMREE without consulting your doctor first.
No, AGAMREE should not be given to children under 4 years of age. The safety and effectiveness of vamorolone have not been studied in this age group. Duchenne muscular dystrophy is typically diagnosed between ages 3 and 5, and corticosteroid treatment is usually initiated once the diagnosis is confirmed and the child meets the clinical criteria for treatment, generally around age 4–6. Your pediatric neurologist or neuromuscular specialist will determine the optimal time to start AGAMREE treatment based on your child’s individual clinical situation.
Clinical evidence suggests that vamorolone (AGAMREE) may have a more favorable effect on bone health compared with prednisone. Traditional corticosteroids like prednisone are well known to cause significant bone loss (osteoporosis) and increase the risk of vertebral fractures, which is a major concern in DMD patients who already have compromised bone density due to reduced mobility and vitamin D deficiency. The dissociative mechanism of vamorolone – reduced trans-activation of genes responsible for bone resorption – is believed to contribute to better preservation of bone mineral density. However, long-term studies are still ongoing, and patients on AGAMREE should continue to have their bone health monitored as recommended by their healthcare team.
References
- European Medicines Agency (EMA). AGAMREE (vamorolone) – Summary of Product Characteristics. Last updated 2025. Available from: EMA EPAR.
- U.S. Food and Drug Administration (FDA). AGAMREE (vamorolone) Prescribing Information. Approved October 2023. Available from: FDA Drug Label.
- Hoffman EP, Riddle V, Siegler MA, et al. Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Steroids. 2018;134:43–52. doi:10.1016/j.steroids.2018.02.010.
- Guglieri M, Bushby K, McDermott MP, et al. Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial (FOR-DMD). JAMA. 2022;327(15):1456–1468. doi:10.1001/jama.2022.4315.
- Mah JK, Clemens PR, Guglieri M, et al. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA Netw Open. 2022;5(1):e2144178. doi:10.1001/jamanetworkopen.2021.44178.
- 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 Neurol. 2018;17(3):251–267. doi:10.1016/S1474-4422(18)30024-3.
- 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 Neurol. 2018;17(4):347–361. doi:10.1016/S1474-4422(18)30025-5.
- World Health Organization (WHO). WHO Model List of Essential Medicines – 23rd List. Geneva: WHO; 2023.
- Guglieri M, Smith EC, Gidaro T, et al. Vamorolone versus prednisone for Duchenne muscular dystrophy (VISION-DMD): a multicentre, randomised, double-blind, active- and placebo-controlled, phase 2b/3 study. Lancet Neurol. 2024. doi:10.1016/S1474-4422(24)00303-0.
- Santhera Pharmaceuticals. AGAMREE (vamorolone) oral suspension – Full Prescribing Information. December 2025.
Editorial Team
This article was written and reviewed by the iMedic Medical Editorial Team, comprising licensed specialist physicians with expertise in neurology, neuromuscular diseases, and clinical pharmacology.
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