Understanding the
Standard of Care
Your roadmap for living well with Duchenne.
What You Need to Know
The words "Standard of Care" can sound intimidating—like something you are supposed to already know, memorize, or somehow get right.
It isn't any of those things.
The Standard of Care is simply the name doctors use for the collection of medical knowledge that guides how people with Duchenne are cared for around the world.
It represents decades of research, clinical experience, and lessons learned from families who have walked this road before you.
Every recommendation exists because doctors, researchers, therapists, and families have continued asking an important question:
"How can we help people with Duchenne live longer, healthier, and fuller lives?"
As those answers have improved, so has the Standard of Care.
It helps families and healthcare teams answer practical questions such which specialists should be involved, which tests should be performed, how often monitoring should happen, which treatments should be discussed, and what can be done today to protect a child's health in the future.
The Standard of Care is not one treatment, one medication, or one doctor's opinion. Think of it as a roadmap. It provides guidance, but it does not predict exactly where your child's journey will lead or require every family to follow the same path.
Some treatments may be appropriate for your child while others are not. Some specialists may become important sooner than others, and some recommendations may not apply for years.
You are not expected to become a medical expert overnight. The Standard of Care gives your family and healthcare team a shared foundation you can ask questions, understand your options, and make informed decisions together—one step at a time.
Every child with Duchenne is different.
The Standard of Care provides the roadmap.
Your healthcare team helps determine the best route for your child.
What the Standard of Care Is — And What It Isn't
The Standard of Care IS
- A roadmap built from decades of research and experience
- A collection of evidence-based recommendations
- A guide that helps healthcare teams make informed decisions
- A framework for protecting your child's health over time
The Standard of Care IS NOT
- A law
- A rulebook
- A checklist every family must complete
- A one-size-fits-all treatment plan
- A replacement for conversations with your healthcare team
Why Does It Matter?
Reduces Variation
The Standard of Care reduces differences in care so children receive consistent, evidence-based support.
Better Planning
It supports families in anticipating needs, preparing for changes, and setting goals.
Shared Knowledge Base
It gives families and healthcare teams a common language and understanding.
Empowered Decisions
It gives you confidence to ask questions, weigh options, and make choices that fit your child and your family.
Right Care, Right Time
It helps children get the right tests, treatments, and monitoring when they need them most.
Standard of Care Map
Newly Diagnosed Stage (Early Diagnosis)
This map shows the key areas of care your child may encounter in the first year after diagnosis. Not every test or appointment happens right away. Your care team will recommend what's right for your child.
Heart
Cardiologist
Duchenne affects the heart muscle over time. Early monitoring helps protect heart function.
- EKG (ECG)
- Echocardiogram
- Cardiac MRI (sometimes)
- Blood tests (troponin, BNP)
- Ejection fraction (EF)
- Cardiomyopathy
- ACE inhibitor / ARB
- Beta blocker
Starting heart-protective medications early may help preserve heart function for the future.
Lungs & Breathing
Pulmonologist
Breathing muscles can weaken over time. Early baselines help guide care.
- Pulmonary Function Test (PFT)
- Sleep study (sometimes)
- Oxygen levels
- End tidal CO₂
- FVC
- Peak cough flow
- Hypoventilation
- BiPAP / CPAP
- Nocturnal support
Early monitoring helps detect changes before symptoms appear and supports better sleep and daytime energy.
Neuromuscular Health
Neurologist
Your neurologist helps monitor muscle strength, motor function, and overall progression.
- Physical exam
- Motor function testing (e.g., NSAA)
- Strength testing
- 6-minute walk test (age appropriate)
- Muscle weakness
- DMD
- DMD gene mutation
- Progression
- Ambulatory status
A baseline helps track changes over time and guides therapies, equipment, and activity planning.
Bone & Endocrine Health
Endocrinologist
Steroid use and reduced mobility can affect bones, growth, and hormones.
- Bone density scan (DEXA)
- Vitamin D level
- Growth measurements
- Hormone evaluation as needed
- Steroids (prednisone / deflazacort)
- Bone density
- Vitamin D
- Growth delay
Protecting bones and supporting growth early reduces fracture risk and supports long-term health.
Nutrition & GI Health
GI Specialist / Dietitian
Digestive problems can affect nutrition, growth, and comfort.
- Weight & BMI tracking
- Stool / GI history
- Swallow evaluation (if needed)
- Labs (as needed)
- Constipation
- GERD (reflux)
- Caloric needs
- Fiber
- Swallow safety
Good nutrition supports growth, energy, and immune health and helps prevent secondary problems.
Cognitive & Emotional Health
Psychologist / Neuropsychologist
Duchenne can affect learning, attention, mood, and emotional well-being.
- Developmental screening
- Neuropsychological evaluation (sometimes)
- Behavioral / emotional assessment
- Executive function
- ADHD
- Anxiety
- Learning support
- IEP / 504 Plan
Early support helps your child succeed in school and build confidence.
Speech, Language & Communication
Speech-Language Pathologist
Some children benefit from support with speech, language, articulation, or swallowing.
- Speech & language evaluation
- Feeding / swallow evaluation (if needed)
- Articulation
- Expressive language
- Receptive language
- Swallow safety
Early support builds communication skills and helps prevent future challenges.
Physical & Occupational Therapy
Therapy PT / OT
Therapy helps maintain movement, flexibility, strength, and independence.
- Range of motion assessment
- Functional assessment
- Equipment evaluation
- Stretching
- Contractures
- Bracing
- Adaptive equipment
- Energy conservation
Early therapy helps protect mobility and supports daily independence.
Routine Health
Primary Care Provider
Primary care helps manage everyday health, vaccines, labs, and general wellness.
- Physical exam
- Routine labs
- Vaccines
- Hearing & vision screenings
- Immunizations
- Growth charts
- Annual checkups
- Well visits
Keeps your child healthy today and supports coordination of care with specialists.
This map is a guide, not a checklist. Your care team will personalize recommendations for your child.
You are not alone. We're here with you.
Your Priorities During the Newly Diagnosed Stage
Focus on what matters most for your family today. These priorities build a strong foundation for the journey ahead.
Understanding
the Diagnosis
Learn what Duchenne is, what to expect, and where to find trustworthy information.
Establishing
Baseline Health
Complete key evaluations and tests to understand your child's current health.
Beginning
Routine Monitoring
Set up regular checkups and screenings to track changes early and stay ahead.
Exploring
Treatment Options
Learn about approved therapies and clinical trials that may be right for your child.
Finding
Your Rhythm
Build routines that support daily life, school, therapies, and family well-being.
Building
Your Care Team
Connect with specialists, therapists, and resources who will support your child and family.