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Understanding Your Child's DMD Mutation and Treatment Eligibility
The exact DMD gene change matters. How deletions, duplications, and point mutations differ, and why the specific mutation shapes eligibility for some treatments.
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#treatments
All articles and blog posts tagged #treatments.
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Heart Medications in Duchenne: Protecting the Heart Before Symptoms
The heart needs protecting in Duchenne, often before symptoms. How medicines like ACE inhibitors and beta blockers help, when they start, and why early matters.
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Making Informed Decisions Together With Your Duchenne Care Team
Duchenne care involves many decisions. How to become an informed partner, understand the options and trade-offs, and share decisions well with the care team.
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Steroid Dosing Schedules in Duchenne: Daily and Intermittent
Steroids in Duchenne can be given on different schedules, from daily to intermittent. How the regimens compare, the evidence, and how the choice is made.
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Managing Daily Medications at Home in Duchenne
Duchenne often means several daily medicines. How to organise doses, avoid missed ones, handle school and travel, track side effects, and keep it manageable.
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Deflazacort vs Prednisone: Choosing a Steroid in Duchenne
Prednisone and deflazacort are the two main steroids for Duchenne. How they compare on benefits and side effects, and how families and teams choose between them.
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Eye Care and Cataract Monitoring on Long-Term Steroids in Duchenne
Long-term steroids in Duchenne raise the risk of cataracts. Why eye monitoring is part of steroid care, what symptoms to watch for, and how often to check.
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Adrenal Insufficiency and Steroid Sick-Day Rules in Duchenne
Long-term steroids in Duchenne can suppress the adrenal glands. What adrenal crisis is, the sick-day rules and stress dosing that prevent it, and when to act fast.
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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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.