About Duchenne Muscular Dystrophy
Key Highlights
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Targets the root genetic cause of DMD, not just symptoms.Offers personalized treatment plans based on the patient's specific genetic mutation.Includes innovative therapies like exon-skipping to promote dystrophin production.Aims to slow disease progression and preserve muscle strength and function.Managed by a specialized multidisciplinary team for comprehensive care.Represents the forefront of precision medicine for neuromuscular disorders./ul
Who is this surgery for?
- Confirmed diagnosis of Duchenne Muscular Dystrophy via genetic testing and clinical evaluation.
- Identification of a specific mutation in the DMD gene that is amenable to targeted therapies like exon-skipping.
- Early intervention, often in childhood, to maximize potential therapeutic benefits.
- As part of a comprehensive management plan including physical therapy and cardiac/respiratory care.
- For patients and families seeking the latest precision medicine options beyond standard supportive care.
How to prepare
- Comprehensive genetic counseling for the patient and family to understand the diagnosis, inheritance, and treatment options.
- Full genetic sequencing and analysis to pinpoint the exact DMD gene mutation.
- Baseline physical assessments including muscle strength, pulmonary function, and cardiac evaluations.
- Multidisciplinary team consultation to develop an integrated care plan.
- Discussion of potential benefits, limitations, and long-term commitment required for genetic therapies.
- Ensuring the patient is medically stable to begin treatment, managing any concurrent health issues.
Risks & possible complications
- Potential side effects specific to the genetic agent used (e.g., injection site reactions, kidney toxicity with some exon-skipping drugs).
- Risk of immune reactions to the viral vectors used in some gene transfer therapies.
- Uncertain long-term efficacy and unknown very long-term safety profile of novel genetic medicines.
- High financial cost and potential challenges with insurance coverage.
- Emotional and psychological impact on the patient and family due to the progressive nature of DMD, even with treatment.
- Not all mutations are currently treatable with available genetic therapies.
Recovery & hospital stay
- Genetic medicine for DMD is typically a chronic, ongoing treatment, not a one-time procedure with a traditional 'recovery' period.
- Regular monitoring is essential, including periodic physical exams, muscle function tests, and safety labs.
- Continued commitment to standard of care is crucial: rigorous physical therapy, respiratory support, and cardiac management.
- Families should maintain a log of any new symptoms or side effects to report to the care team.
- Outpatient follow-up appointments are scheduled frequently (e.g., every 3-6 months initially) to assess progress and adjust the care plan.
- Psychological support and connection with patient advocacy groups are highly recommended for ongoing coping.
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Typical hospital stay: Typically outpatient / 0 days (for infusions or evaluations, may require day-care)
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Expected recovery time: Ongoing chronic treatment; initial adaptation period of 2-4 weeks post-treatment initiation
Frequently Asked Questions
If you are considering duchenne muscular dystrophy in India, these questions and answers can help you make a confident, informed decision.
Top-rated hospitals in India with genetic medicine departments and experienced surgeons are ideal for this procedure. Use MediFyr to compare facilities, reviews, and doctor profiles before you decide.
Look at the doctor’s years of experience, hospital association, patient reviews, and how often they perform duchenne muscular dystrophy. MediFyr helps you compare genetic medicines and book consultations online.
The overall cost depends on hospital category, surgeon’s experience, room type, implant or device used (if any), length of stay, tests, and post-operative care. Our team can help you get cost estimates from multiple hospitals before you decide.
Procedure cost in other countries
Here is an overview of how the estimated cost, hospital stay, and recovery time for duchenne muscular dystrophy compare across other countries where we have data.
| Country | Estimated cost range | Typical stay | Recovery time | View details |
|---|---|---|---|---|
| India | INR 500 Thousand – 2.5 Million | Typically outpatient / 0 days (for infusions or evaluations, may require day-care) | ~ Ongoing chronic treatment; initial adaptation period of 2-4 weeks post-treatment initiation | Know More |
| Turkey | TRY 1.75 Million – 8.75 Million | Typically outpatient / 0 days (for infusions or evaluations, may require day-care) | ~ Ongoing chronic treatment; initial adaptation period of 2-4 weeks post-treatment initiation | Know More |
Myra Kumar is a 28-year-old high...
Myra Kumar is a 28-year-old high school biology teacher from a close-knit family with no prior known history of neuromuscular disorders. Her symptoms began subtly in her late teens with frequent tripping and difficulty keeping up in gym class, which she attributed to clumsiness. By her mid-twenties, she experienced progressive muscle weakness in her hips and thighs, making stairs challenging and rising from chairs difficult. After years of inconclusive tests, a neurologist, noting her elevated creatine kinase levels and characteristic calf hypertrophy, suspected Duchenne Muscular Dystrophy (DMD), highly unusual for a female. The doctor recommended genetic testing to confirm the diagnosis, specifically a multiplex ligation-dependent probe amplification (MLPA) test to analyze the DMD gene for deletions or duplications. Myra underwent a blood draw for the test. The experience was physically simple but emotionally heavy, as she waited weeks for the results. The test confirmed a diagnosis of symptomatic DMD carrier with a significant mutation, explaining her manifesting symptoms. The outcome was not a recovery, but a definitive answer. It allowed her to transition from a frustrating diagnostic odyssey to a clear management plan involving cardiac and respiratory monitoring, physical therapy, and genetic counseling for family planning. Emotionally, she moved from years of anxiety and self-doubt, feeling like her body was betraying her without explanation, to a painful but solid ground of understanding. While grieving the loss of her previous physical certainty, she felt empowered with knowledge and a path forward, allowing her to advocate for herself and make informed life decisions.
Genetic Medicines for Duchenne Muscular Dystrophy
Explore experienced genetic medicines who regularly perform duchenne muscular dystrophy and provide pre- and post-operative care in India.
- 16 Years Experience
- Genetic Medicine
Miot Hospital Chennai, Chennai
- 14 Years Experience
- Genetic Medicine
Miot Hospital Chennai, Chennai
- 4 Years Experience
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Miot Hospital Chennai, Chennai