What Are the Treatments for Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) is the kind of diagnosis that turns ordinary calendar planning into
“Okay, but what does the care plan look like next month… and next year… and next decade?”
The good news (and yes, we’re allowed to use that phrase here) is that DMD care has changed dramatically:
there are more FDA-approved therapies than ever, and there’s a well-mapped playbook for protecting
muscles, heart, lungs, bones, and quality of life.

The realistic news: there still isn’t a cure. But modern treatment is not “do nothing and hope.”
It’s a strategic, layered approach that can slow progression, preserve function longer, and help people
with Duchenne live fuller lives. Think of it like building a house in hurricane season:
you reinforce every wall you can, earlybecause waiting until the storm arrives is not a vibe.

First, What Does “Treatment” Mean in Duchenne?

Treatments for Duchenne muscular dystrophy fall into two big buckets:

• Disease-modifying therapies: medications designed to slow DMD’s progression or address the underlying biology
  (like inflammation, muscle breakdown, or dystrophin production).
• Supportive care: the everyday (but powerful) interventions that protect joints, lungs, heart, bones, and independence
  including physical therapy, respiratory support, cardiac meds, mobility equipment, and surgery when needed.

The best outcomes usually come from a multidisciplinary neuromuscular care teamneurology, cardiology,
pulmonology, physical therapy, orthopedics, nutrition, endocrinology, and mental health support. Not because
doctors love group projects (they don’t), but because Duchenne affects multiple systems and the “weakest link”
can change over time.

FDA-Approved Therapies Specific to Duchenne (Plus the Classics)

In the U.S., there are several FDA-approved treatments specifically for DMD. Some work for many patients;
others are mutation-specific (meaning genetic testing determines eligibility). Your neuromuscular specialist will
match options to age, stage, mutation type, and overall health.

1) Corticosteroids: Still the Backbone of DMD Treatment

Corticosteroids have been the “workhorse” of Duchenne care for years because they can slow muscle weakness
and help preserve function. They’re not magic, but they’re meaningful. In the U.S., two steroid-based options are FDA-approved for DMD:

• Deflazacort (EMFLAZA) – approved for patients age 2 and older.
• Vamorolone (AGAMREE) – approved for patients age 2 and older; designed to keep benefits while potentially reducing certain side effects compared with traditional steroids.

Many clinics also use prednisone/prednisolone (widely used in DMD care) depending on the patient and provider preference.
The dosing schedule (daily vs. intermittent), timing of start, and which steroid to use are individualized.

What steroids can help with:

• Slowing loss of strength and motor function
• Extending the time a child can walk independently
• Reducing inflammation-related muscle damage
• Delaying scoliosis progression in some patients
• Supporting heart and breathing function over time as part of a full care plan

What to watch closely: steroids require real monitoring, not “hope and vibes.” Common concerns include:

• Weight gain and appetite changes
• Behavior and mood changes (especially early on)
• Bone thinning (osteoporosis) and fracture risk
• Growth delay and puberty-related issues
• Cataracts (reported more with some steroid regimens)
• Higher infection risk; vaccine timing matters

Practical tip: families often find it helpful to treat steroid side effects like a parallel project
nutrition support, vitamin D/calcium guidance, bone density tracking, and mental health check-ins
because “we’ll deal with it later” tends to show up with receipts.

2) DUVYZAT (Givinostat): A Nonsteroidal Option for Many Genetic Variants

DUVYZAT (givinostat) is an FDA-approved oral medication for Duchenne in patients age 6 and older.
It’s a histone deacetylase (HDAC) inhibitor, and while the exact mechanism in DMD isn’t fully pinned down in a neat bow,
it’s intended to reduce harmful processes like inflammation and muscle degeneration.

Why this matters: unlike exon-skipping therapies, givinostat is not limited to a single exon mutation group.

Monitoring and side effects: DUVYZAT can affect labs and GI tolerance, so clinicians commonly monitor:

• Platelets (risk of low platelet count)
• Triglycerides
• Digestive symptoms like diarrhea or nausea

3) Exon-Skipping Therapies: Mutation-Specific “Reading Frame” Workarounds

DMD is caused by changes in the dystrophin (DMD) gene. Some mutations disrupt the genetic “reading frame.”
Exon-skipping therapies use antisense technology to “skip” a specific exon during RNA processing,
potentially restoring the reading frame so the body can make a shorter, more functional dystrophin-like protein.

Translation: it’s like editing a sentence by removing a broken word so the rest of the sentence makes sense again.
Not perfect, but better than total gibberish.

FDA-approved exon-skipping therapies in the U.S. include:

• EXONDYS 51 (eteplirsen) – for mutations amenable to exon 51 skipping
• VYONDYS 53 (golodirsen) – for exon 53 skipping
• VILTEPSO (viltolarsen) – also for exon 53 skipping
• AMONDYS 45 (casimersen) – for exon 45 skipping

What to know before getting excited (or overwhelmed):

• Eligibility depends on genetic testing. A neuromuscular clinic will usually confirm the mutation type and whether it’s “amenable.”
• These are typically ongoing IV infusions. Families plan around infusion schedules like they’re a second job.
• Approvals have often involved surrogate markers (like dystrophin production). Functional benefit is still an area of continued study.

4) Gene Therapy (ELEVIDYS): One-Time Infusion, Big Promise, Serious Safety Monitoring

ELEVIDYS (delandistrogene moxeparvovec) is a gene therapy designed to deliver a micro-dystrophin gene to muscle cells.
It’s given as a single intravenous infusion, with the goal of enabling the body to produce micro-dystrophin.

Important update for readers: as of November 14, 2025, U.S. labeling changes added a Boxed Warning
for risk of serious liver injury and limited the approved indication to ambulatory (walking) patients age 4 and older
with a confirmed DMD gene mutation. (If you read older articles saying “ambulatory and non-ambulatory,” that’s exactly why checking dates matters.)

What families should expect with gene therapy discussions:

• Eligibility screening (age, walking status, mutation confirmation, and other clinical criteria)
• Immune management (often involving steroids or other protocols around infusion)
• Frequent lab monitoring after infusion, especially liver function tests
• Realistic expectations: gene therapy is a major step forward, but it’s not a rewind button to “before Duchenne.”

Supportive Treatments That Protect Function (A Lot More Than People Think)

If disease-modifying therapies are the headline, supportive care is the entire plot. It’s what helps a child keep
moving safely, protects the heart and lungs, and prevents “small problems” from turning into big emergencies.

Physical Therapy, Stretching, and Smart Activity

Physical therapy (PT) in Duchenne isn’t about turning someone into a superheroit’s about preserving range of motion,
preventing contractures, and keeping movement as efficient as possible. Many care teams recommend:

• Regular PT evaluations and home stretching routines
• Night splints or braces when appropriate
• Gentle strengthening and low-impact activities (often with careful pacing)
• Avoiding “overwork weakness” from exhausting, high-resistance workouts

Real-life example: a child who starts toe-walking may benefit from targeted stretching, ankle-foot orthoses, and gait support
before it becomes a pain-and-falls situation. It’s not glamorous, but it’s effective.

Orthopedic Care: Bracing, Contracture Management, and Scoliosis Treatment

As muscles weaken, joints can tighten and the spine can curve. Orthopedic management may include:

• Bracing and positioning supports
• Standing devices (when recommended)
• Surgery for contractures in selected situations
• Scoliosis monitoring and, when needed, spinal surgery (often coordinated carefully with respiratory status)

This is also where anesthesia planning matters. People with neuromuscular conditions can have special considerations during surgery,
so procedures are typically coordinated with experienced teams.

Cardiac Treatment: Protect the Heart Early, Not After It Complains

Duchenne affects cardiac muscle, and cardiomyopathy can develop even before obvious symptoms show up.
Standard care often includes:

• Cardiology evaluation at diagnosis and regular follow-ups
• Echo and/or cardiac MRI monitoring
• Starting heart-protective medicines (commonly ACE inhibitors or ARBs)often around age 10 even if function looks normal,
  or earlier if changes appear
• Adding beta-blockers or other heart failure therapies when indicated

Example “why this matters”: a child might feel fine, but imaging can show early cardiac changes.
Starting medication early is a prevention strategylike putting on sunscreen before you fry, not after.

Respiratory Care: Sleep, Cough Strength, and Ventilation Support

Respiratory muscles gradually weaken in DMD, and breathing support can become necessary over time.
Good respiratory care is proactive:

• Regular pulmonary function testing (more frequent as the disease advances)
• Sleep evaluation if symptoms suggest sleep-disordered breathing (snoring, morning headaches, daytime sleepiness)
• Cough-assist devices to help clear secretions when cough becomes weak
• Noninvasive ventilation (often at night first) when indicated
• Vaccines and infection planning (because respiratory infections can hit harder)

Families often say respiratory equipment feels intimidatinguntil it becomes the thing that helps energy,
sleep quality, and fewer hospital trips. In other words: scary in the brochure, comforting in real life.

Bone Health, Growth, and Nutrition

Steroids plus reduced mobility can increase osteoporosis risk. Care teams frequently monitor and support:

• Vitamin D and calcium intake (food first when possible)
• Bone density screening when indicated
• Fall prevention and fracture planning
• Healthy weight support (balancing steroid appetite with nutrition needs)
• Endocrine issues (growth, puberty, and steroid-related metabolic effects)

Learning, Behavior, and Mental Health Supports

DMD can be associated with learning differences and attention challenges, and the mental load on families is real.
Helpful supports may include:

• Neuropsychological evaluation when needed
• School accommodations (IEP/504 plans)
• Counseling for anxiety, depression, or adjustment stress
• Social work support for equipment, insurance, and family logistics

A Practical Treatment Roadmap by Stage

Duchenne care changes over time, but planning by “stage” can make it feel less like chaos and more like strategy.
A simplified roadmap often looks like this:

Early ambulatory years

• Start disease-modifying therapy discussions (often steroids)
• Establish PT/stretching routines early
• Baseline cardiology and pulmonary evaluations
• Genetic testing to identify mutation-specific options

Late ambulatory years

• Adjust meds and manage steroid side effects aggressively
• Increase monitoring of heart and lung function
• Orthopedic supports for gait, falls, and contractures
• Plan early for mobility equipment (before it’s urgent)

Early non-ambulatory years

• Upper-limb function and seating/positioning become major priorities
• Respiratory support discussions often become more active
• Cardiac medications may be added or adjusted
• Bone health and fracture prevention become even more important

Late non-ambulatory years

• More frequent respiratory monitoring and ventilation support as needed
• Ongoing cardiac management
• Swallowing/nutrition planning when indicated
• Focus on comfort, independence, communication, and quality of life goals

Clinical Trials and Future Directions

DMD research is moving fast. Clinical trials continue to explore:

• Next-generation exon-skipping (more efficient delivery and broader mutation coverage)
• Gene editing approaches aiming to correct mutations at the DNA level (still investigational)
• Muscle growth and anti-fibrotic strategies to preserve muscle quality
• Cardiac-focused therapies targeting Duchenne-related cardiomyopathy

If your clinic offers trial screening, it can be worth askingeven if you don’t join. Understanding what’s in the pipeline
helps you plan and keeps you from relying on outdated social media rumors (which, medically speaking, are the worst roommates).

Questions to Ask Your Neuromuscular Team

• Which FDA-approved DMD therapies are appropriate for this mutation and age?
• What are the goals of treatment right nowstrength, walking, heart protection, breathing, comfort?
• How will we monitor side effects (weight, mood, bones, labs, liver function, heart imaging, lung tests)?
• What PT/stretching plan should we do at homeand how often should we reassess?
• When should we start or adjust heart medications?
• When should we consider respiratory equipment like cough-assist or nighttime ventilation?
• Are there clinical trials we should learn about this year?

Conclusion

Treatments for Duchenne muscular dystrophy are no longer limited to “supportive care only.”
In the U.S., FDA-approved options include steroid-based therapies (like deflazacort and vamorolone),
a nonsteroidal disease-modifying therapy (givinostat), mutation-specific exon-skipping medications,
and gene therapy for eligible ambulatory patientsalongside a deep bench of supportive strategies that protect the heart,
lungs, bones, and daily independence.

The most effective approach is layered: treat what you can treat now, monitor what you can monitor early, and plan before you’re forced to.
And yesthere’s room for hope here. Not the fluffy kind. The practical kind that shows up to clinic visits with questions,
a spreadsheet, and maybe snacks.

Experiences: What Treatment for Duchenne Can Feel Like in Real Life (About )

If you ask families what treating Duchenne muscular dystrophy is “like,” you’ll rarely get a single answerbecause it changes by stage,
by personality, by side effects, and by what a family can realistically carry. But there are patterns that show up again and again,
and they’re worth naming because they make people feel less alone.

Early on, many parents describe a strange mix of urgency and disbelief. The first big treatment conversationsoften about starting steroids
can feel like being asked to choose between two hard things: the disease you can see coming versus side effects you might see sooner.
Some families say the first months on steroids are the “adjustment season,” where appetite jumps, moods can swing, and routines change.
At the same time, they often notice functional benefits that make the effort feel worthwhilelike getting up from the floor more easily,
climbing stairs with less struggle, or simply having more stamina for regular kid stuff.

The day-to-day reality is that supportive care becomes a quiet hero. Stretching at home sounds simple until you’re doing it consistently
after school, before dinner, and again on a weekend when everyone is tired. Families often learn that the “best” plan isn’t the fanciest plan
it’s the one that actually gets done. A 10-minute routine that happens five days a week can be more powerful than a 45-minute routine
that happens twice a month and makes everyone cry. (No judgment. It happens.)

As care becomes more medicalcardiology visits, pulmonary testing, infusion schedules for mutation-specific therapiesfamilies often become
experts in scheduling, paperwork, and advocating. People describe learning a new language made of acronyms: PFTs, MRI, AFOs, IEPs.
It can be exhausting, but there’s also a sense of empowerment that grows when families realize: “We’re not just reacting. We’re steering.”

Gene therapy conversations can bring a different emotional weight. Many families talk about hope that feels huge and fragile at the same time,
especially as eligibility rules and safety monitoring evolve. The most grounded families tend to treat it like a serious medical decision,
not a miracle auditionasking about risks, liver monitoring, what “success” looks like, and how the rest of the care plan continues afterward.
Because even with breakthrough therapies, physical therapy doesn’t stop, heart care doesn’t stop, and life still needs structure.

Perhaps the most consistent “experience” families describe is this: progress is rarely one dramatic moment. It’s dozens of small wins
a better night’s sleep after respiratory support, fewer falls after bracing changes, a steadier routine after medication adjustments,
or a school plan that finally clicks. Over time, many families say they stop chasing perfection and start chasing momentum.
And in Duchenne care, momentum is powerful.