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What are the Therapies to Treat Muscular Dystrophy in Children?

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therapies to treat muscular dystrophy
  • November 14, 2024

What are the Therapies to Treat Muscular Dystrophy in Children?

Therapies to treat muscular dystrophy

Muscular dystrophy (MD) is a group of genetic diseases causing muscle weakness and degeneration. Among children, Duchenne Muscular Dystrophy (DMD) is the most common, affecting mainly boys and typically diagnosed between ages 3 and 5. As MD is progressive, symptoms worsen over time, making early intervention crucial. Though there’s currently no cure, various therapies can improve the quality of life and manage symptoms. Here’s an overview of the main therapies for treating muscular dystrophy in children.

Therapies to treat muscular dystrophy in children

1. Physical Therapy (PT)

Physical therapy is foundational in managing MD and focuses on preserving mobility and muscle strength. Key techniques include:

Stretching and Range-of-Motion Exercises: These prevent contractures, which are the shortening and hardening of muscles that reduce flexibility and increase stiffness in joints.

Strengthening Exercises: Carefully supervised, low-impact exercises can help maintain muscle strength without overexerting the muscles.

Breathing Exercises: These are crucial as MD progresses since respiratory muscles weaken over time.

2. Occupational Therapy (OT)

Occupational therapy helps children adapt to daily activities, maximizing independence. Therapists assess each child’s needs and provide adaptive techniques and tools for daily living tasks, like eating, dressing, and using mobility devices.

3. Speech and Respiratory Therapy

Speech Therapy: Some types of MD, especially as they progress, affect facial and throat muscles, leading to speech difficulties. Speech therapists work with children to improve articulation, speech clarity, and communication techniques.

Respiratory Therapy: Respiratory muscles weaken as MD progresses, leading to difficulties with breathing and coughing. Respiratory therapists provide breathing exercises and, in some cases, devices that help assist breathing, especially at night.

4. Medication Therapy

Medications play a role in slowing disease progression and managing symptoms. Some common medications used in MD therapy include:

Corticosteroids (e.g., prednisone, deflazacort): These can slow muscle degeneration and preserve muscle function for longer periods. However, they have side effects such as weight gain, weakened bones, and high blood pressure, which require careful management.

Exon Skipping Therapy: A newer approach, exon skipping uses synthetic drugs (like eteplirsen) to help cells produce more functional dystrophin, the protein missing in DMD. This therapy targets specific genetic mutations and is effective only in certain types of MD.

Heart Medications: Since MD often affects the heart muscles, drugs like ACE inhibitors or beta-blockers are used to manage heart complications.

5. Gene Therapy

Gene therapy aims to address the root cause of MD by targeting the genetic mutations responsible. Research is ongoing, and this field shows promise, with recent clinical trials aiming to deliver healthy copies of genes or replace faulty ones. Although not yet widely available, gene therapy could provide a long-term solution for MD and potentially stop its progression.

6. Stem Cell Therapy

Stem cell therapy is another experimental treatment that aims to regenerate muscle tissue by introducing healthy muscle cells. Stem cells may be injected into muscles or genetically modified to increase their effectiveness. Though still in the early stages, stem cell therapy is promising and has shown encouraging results in some trials.

7. Assistive Devices and Mobility Aids

Various assistive devices can significantly improve the quality of life for children with MD. These include:

Braces and Orthoses: These helps stabilize joints, support muscles, and improve walking ability for as long as possible.

Wheelchairs and Mobility Devices: As MD progresses, children may need power wheelchairs to maintain mobility and independence.

Cough Assist Devices: For children with weakened respiratory muscles, these devices help them cough more effectively and avoid respiratory infections.

8. Nutritional Support

Nutrition plays a role in managing MD symptoms, especially for children taking corticosteroids. A balanced diet rich in calcium and vitamin D helps maintain bone health and manage weight, which can be challenging due to reduced physical activity.

9. Supportive and Psychological Therapies to treat muscular dystrophy

Living with an MD can be emotionally challenging. Psychological therapy, such as counseling, can help children cope with their condition and support their mental health. Support groups provide a sense of community, allowing children and families to connect with others facing similar challenges.

10. Clinical Trials and Emerging Therapies

Many promising therapies for MD are currently under investigation. Clinical trials provide access to cutting-edge treatments, like CRISPR-based gene editing and newer gene therapies, which could potentially modify or correct genetic mutations responsible for MD.

Bottom Line

While a cure for muscular dystrophy is still out of reach, ongoing advancements in therapies to treat muscular dystrophy continue to improve outcomes for children with MD. Comprehensive treatment plans that include physical, occupational, and respiratory therapies, alongside medication, assistive devices, and emotional support, are vital. If you’re a parent of a child with MD, then you can reach out to us or visit Dr. Habib’s Foster CDC for a comprehensive evaluation of your child’s condition and appropriate treatment plan.

Staying informed on the latest research and working closely with specialists and therapists at FOSTER CDC can help you provide the best care and support.

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