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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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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