Article
Duchenne, Drug Approval, and Public Policy: Why Georgian Families Are Demanding Action
How regulators, payers, and politics meet in the Duchenne treatment access dispute in Georgia, and what it says about rare disease policy.
Editorial byline
Helena Marsh
Helena Marsh writes research-based summaries covering standards of care, regulatory decisions, and family resources for Duchenne muscular dystrophy. Every claim is linked to a primary source such as the FDA, EMA, PubMed, or recognised patient organisations. Posts are informational and do not replace consultation with a qualified care team.
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Article
Children Waiting While Time Runs Out: The Human Cost of Duchenne in Georgia
Inside the Duchenne movement in Georgia: families, children, daily life, and what it costs to wait.
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Female Carriers and Cardiomyopathy in Duchenne Muscular Dystrophy
Female carriers of DMD: manifesting carriers, cardiac involvement, why surveillance matters even for asymptomatic mothers, and what to ask the team.
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Insurance Appeals for Duchenne Families
Insurance appeals in DMD: when denials happen, how to structure the appeal letter, what evidence helps, and how patient organizations support families.
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Muscle MRI in Duchenne Muscular Dystrophy
Muscle MRI in DMD: fat fraction, T2 mapping, Mercuri grading. How imaging biomarkers track disease beyond cardiac and what they add to functional tests.
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Preparing for Surgery in Duchenne Muscular Dystrophy
Surgery preparation in DMD: pre-operative assessments, multidisciplinary planning, family logistics, and what to bring to the hospital.
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Patient Registries and Natural History Studies in DMD
DMD patient registries: CINRG, Duchenne Registry, TREAT-NMD, MD STARnet. What they do, who can join, and why enrollment supports the whole community.
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Telemedicine and Remote Care in Duchenne Muscular Dystrophy
Telemedicine in DMD: when virtual visits work, what they cannot replace, and how families can prepare to get the most from a remote appointment.
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The DMD Research Pipeline: What's Beyond Approved Therapies
DMD research pipeline: CRISPR gene editing, next-generation gene therapy, exon skipping advances, anti-fibrotic approaches, and what families should know.
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Working With DMD Patient Organizations
Patient organizations in DMD: PPMD, MDA, CureDuchenne, Jett Foundation, Duchenne UK. What they do, how families use them, and how to get involved.
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Cardiac MRI in Duchenne Muscular Dystrophy
Cardiac MRI in DMD: late gadolinium enhancement, early fibrosis detection, when it is used, and why it changes the cardiac surveillance plan.
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Managing Illness Days at Home in Duchenne Muscular Dystrophy
Managing sick days in DMD at home: fever, respiratory infections, missed corticosteroids, and when to call the clinic or head to the emergency room.
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Functional Assessments in Duchenne Muscular Dystrophy
Functional assessments in DMD: 6MWT, NSAA, time-to-stand, PUL. What each measures, why they matter for trials and routine care.
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Siblings of Children With Duchenne Muscular Dystrophy
Siblings in DMD families: what they often experience, what helps, and how parents and care teams can support them through the long course of the disease.
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Friendships and Social Life for Children With Duchenne
Social life in DMD: peer relationships, summer camps, hobbies, and what families can do to keep a child connected as the disease progresses.
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Vaccinations in Duchenne Muscular Dystrophy
Vaccinations in DMD: influenza, pneumococcal, COVID-19, why respiratory protection matters more, and what to discuss with the care team.
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Adult Care in Duchenne Muscular Dystrophy
Adult care in DMD: ongoing cardiac, respiratory, endocrine, and rehab needs for patients living into their 20s, 30s, and beyond.
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Returning to School After a Duchenne Diagnosis
Returning to school after a DMD diagnosis: briefing teachers, talking to classmates, the first weeks, and what families can plan.
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Endocrine Care in Duchenne Muscular Dystrophy
Endocrine care in DMD: growth, puberty, adrenal suppression, and why patients on long-term corticosteroids need an endocrinologist on the team.
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Financial Planning and Insurance for Duchenne Families
Practical guidance on insurance, disability benefits, copay assistance, equipment funding, and long-term financial planning for DMD families.
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The DMD Care Team: Who Does What
A practical guide to the multidisciplinary DMD care team: neuromuscular, cardiac, pulmonary, rehab, endocrine, psychosocial, and how they coordinate.
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Speech and Language Therapy in Duchenne Muscular Dystrophy
Speech-language therapy in DMD: dysphagia assessment, voice preservation, communication strategies, and when to start working with an SLP.
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Bowel and Bladder Care in Duchenne Muscular Dystrophy
Bowel and bladder dysfunction in DMD: prevalence, why it happens, practical management, and when to escalate.
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Genetic Counseling for Duchenne Families
Genetic counseling in DMD: carrier testing for mothers and sisters, recurrence risk, family planning options, and what a counseling visit covers.
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Assistive Technology for Communication and Daily Life in DMD
Assistive technology in DMD: switches, eye-gaze, AAC, smart-home, and how to stage the toolkit as upper-extremity function changes.
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Sleep in Duchenne Muscular Dystrophy
Sleep in DMD: why night breathing changes first, polysomnography, what overnight oximetry misses, and when to start ventilation.
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Dental Care for Duchenne Families
Dental care in DMD: routine cleaning, oral health, sedation and anesthesia precautions, and how to brief a dentist who has not treated DMD before.
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Pain Management in Duchenne Muscular Dystrophy
Chronic pain in DMD is common, under-recognized, and treatable. A practical overview for families and care teams.
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Scoliosis in Duchenne Muscular Dystrophy
Scoliosis in DMD: why curves develop, what surveillance involves, when surgery is considered, and what families can ask the team.
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Travel With Duchenne: A Practical Guide for Families
Travel with DMD: airline notice for power wheelchairs, batteries, BiPAP and cough assist, medications, documents, and what to plan before the trip.
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Emergency Preparedness for Duchenne Families
Emergency preparedness in DMD: the emergency card, steroid stress dosing, anesthesia and cardiac alerts, and how to brief a hospital that does not know your child.
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Nutrition in Duchenne Muscular Dystrophy
Nutrition in DMD: weight management on steroids, bone-supporting nutrients, constipation, swallowing changes, and when to involve a dietitian.
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Anesthesia Safety in Duchenne Muscular Dystrophy
Anesthesia in DMD requires specific precautions: succinylcholine is contraindicated, volatile agents are avoided, and pre-op cardiac and respiratory assessment is essential.
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Home Accessibility for Duchenne Families
Practical home modifications for DMD families: doorways, bathrooms, bedrooms, ramps, lifts, and how to plan ahead without overbuilding.
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Physical Therapy in Duchenne Muscular Dystrophy
Physical therapy in DMD: stretching, contracture prevention, what activities help, and which to avoid. A practical guide for families.
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Choosing a Wheelchair in Duchenne Muscular Dystrophy
Wheelchair selection in DMD: when to consider one, manual vs power, seating, growth-ready features, and what families should ask the team.
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Bone Health in Duchenne Muscular Dystrophy
Bone health in Duchenne muscular dystrophy: fracture risk, DEXA scans, vitamin D, calcium, and what families and clinicians can do.
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Respiratory Care in Duchenne Muscular Dystrophy
Respiratory care in Duchenne muscular dystrophy: lung function decline, sleep-disordered breathing, non-invasive ventilation, cough assist, and surveillance.
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Heart Care in Duchenne: Why Monitoring Starts Early
DMD cardiomyopathy is now the leading cause of death in Duchenne. Early heart monitoring and cardio-protective therapy are core to current standards of care.
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DMD Patient Advocacy History and Impact
Dmd patient advocacy history shows how families shaped research priorities, drug review, care standards, and access debates.
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Vamorolone (Agamree): A Modified Steroid for DMD
Vamorolone is a glucocorticoid receptor modifier approved for Duchenne. It aims to preserve anti-inflammatory benefit while reducing some side effects.
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Reading Health Information About DMD Carefully
Health information about Duchenne can be misleading, exaggerated, or incomplete. Practical media literacy questions for families.
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Givinostat (Duvyzat): First Non-Steroidal DMD Treatment
Givinostat, marketed as Duvyzat, is the first non-steroidal DMD drug approved by the FDA. What the science, the trial, and the label actually say.
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Newborn Screening for Duchenne: Pros and Limits
Newborn screening for Duchenne may support earlier care and counseling, but evidence, ethics, and treatment access remain unsettled.
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Elevidys Explained: The First DMD Gene Therapy
Elevidys is the first FDA-approved gene therapy for Duchenne muscular dystrophy. What it is, who it is for, and what the regulatory record actually says.
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Talking to a Child About a Progressive Disease
Age-appropriate, honest conversations about Duchenne with the child who has it: what helps, what to avoid, and when to ask for help.
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From Parent to Advocate: A Practical Guide
Many DMD parents become advocates. This guide describes what that work involves, how to start, and how to do it sustainably.
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DMD Treatment Access by Country: Why It Varies
Dmd treatment access by country varies through regulators, payers, health technology assessment, pricing, and local budgets.
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Crossing Borders for Duchenne Treatment
Some families relocate or travel abroad for Duchenne treatment. The decision involves access, evidence, logistics, finances, and family.
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DMD Transition to Adulthood: Planning Ahead
Dmd transition to adulthood covers adult care, independence, assistive technology, education, work, and identity.
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How Duchenne Reshapes Family Roles
Duchenne muscular dystrophy quietly redraws who does what at home, with consequences for partners, siblings, and grandparents.
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The First Year After a Duchenne Diagnosis
The first year after a Duchenne diagnosis is intense, fragmented, and full of decisions. A guide to what families can expect and ask.
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Mental Health Duchenne Muscular Dystrophy Guide
Mental health in Duchenne muscular dystrophy care: patients, parents, siblings, anxiety, depression, behavior, and caregiver strain.
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Caregiver Burnout in Duchenne Families
Caregiver burnout in Duchenne muscular dystrophy is common, predictable, and treatable. Recognizing the signs early protects the whole family.
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School Accommodations Duchenne Families Can Discuss
School accommodations for Duchenne can cover access, fatigue, learning, emergency planning, mobility, and inclusion in everyday school life.
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How to Read Regulatory News About DMD
Regulatory news about DMD therapies can be confusing. A practical guide to reading FDA, EMA, and post-marketing safety updates carefully.
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DMD Standards of Care: What Good Care Includes
Dmd standards of care include coordinated neuromuscular, heart, lung, rehab, bone, nutrition, and psychosocial support.
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DMD Clinical Trial: A Family Reading Guide
Use this dmd clinical trial guide to understand phases, endpoints, eligibility, placebo, risks, and questions for a care team.
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Health Technology Assessment in Rare Disease
Health technology assessment shapes which rare disease therapies are funded, and how. A primer for families and advocates.
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DMD Treatment Cost: Why Prices Are So High
Understand dmd treatment cost factors, including rare disease economics, manufacturing, evidence uncertainty, and payer decisions.
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Accelerated Approval and Rare Disease Therapy
Accelerated approval pathways are central to modern rare disease therapy. They are useful, controversial, and often misunderstood.
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Gene Therapy Duchenne Muscular Dystrophy Update
Gene therapy for Duchenne muscular dystrophy, updated: micro-dystrophin, FDA approval, safety signals, and the unresolved questions ahead.
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Exon Skipping Duchenne Therapies Explained
Exon skipping for Duchenne, explained: who may qualify, why approvals differ across jurisdictions, and why evidence debates continue.
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Corticosteroids in Duchenne Muscular Dystrophy
Corticosteroids in Duchenne muscular dystrophy: evidence on benefits, side effects, and the treatment questions families can bring to clinicians.
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DMD Diagnosis: From Suspicion to Confirmation
Understand the dmd diagnosis pathway, from early symptoms and CK testing to genetic confirmation and specialist follow-up.
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Early Signs of Duchenne Muscular Dystrophy
Early signs of Duchenne muscular dystrophy include delayed walking, frequent falls, calf enlargement, and when to ask a clinician about testing.
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DMD Carrier Mother: What to Know
A dmd carrier mother may need genetic counseling, family testing, and cardiac follow-up. Learn the questions to raise with clinicians.
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The Dystrophin Gene Explained Simply
This dystrophin gene explained guide covers what dystrophin does, how mutations disrupt muscle, and why results shape DMD care.
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Duchenne vs Becker Muscular Dystrophy
Duchenne versus Becker muscular dystrophy in plain English: the shared gene, the different severity, and the diagnosis questions families ask.
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What Is Duchenne Muscular Dystrophy?
What is Duchenne muscular dystrophy? Why it affects muscle, how symptoms appear, and what families can ask their care team next.