Treatment Options for Limb-Girdle Muscular Dystrophy That Make a Difference
As a parent, nothing prepares you for the moment when your child is diagnosed with a condition like Limb-Girdle Muscular Dystrophy (LGMD).
It is obvious to have many questions in your mind regarding your child’s health, well-being, and future. Questions like “What does this mean for my child?” “Will they be able to live a normal life?” and “Is there a treatment?” are common.
We are helping you to find out the answers to each of these questions here. You will understand what LGMD is, what signs to look out for, and how you can support your child to get a life where they can perform everyday tasks without discomfort and live an independent life.
What is Limb-Girdle Muscular Dystrophy?
LGMD is a group of inherited muscle disorders that cause progressive muscle weakness in the muscles around the hip and shoulder. A person with this disorder has difficulty in movement, as these muscles are responsible for movements like walking, lifting, and climbing stairs. The affected person may find all these activities challenging.
LGMD is not a single disease, and it is a family of genetic disorders with many subtypes. The progression and severity of all these subtypes vary. But all forms have one sign in common: a gradual decline in muscle strength.
What are the Symptoms of Limb-Girdle Muscular Dystrophy (LGMD)?
For accurate diagnosis and effective treatment, understanding LGMD plays a vital role. Let’s discuss some of the main characteristics, signs, and symptoms:
LGMD primarily weakens muscles in the hips, thighs, and shoulders.
There is a progression of deterioration in the lower limbs and subsequently in the upper limbs also.
The time of onset and severity of symptoms can be different even in those carrying the same mutation.
Feeding difficulties may be preceded or accompanied by poor walking ability, a “waddling gait,” and weakness in the hips and legs, which are some of the early signs.
Patients have significant difficulty walking and moving, climbing stairs, and getting up from the floor. They often need mobility aids for all these tasks. This often includes using a full-time wheelchair or other mobility aids.
Due to shoulder weakness, they experience an inability to lift arms, carry heavy objects, or reach overhead.
Some forms of LGMD affect the muscles in the face, making speech and swallowing hard. Muscle weakness can cause heart failure and breathing difficulties. It can typically start on both sides of the body almost at the same rate.
Patients may experience musculoskeletal disability, in which painful stiffness, cramping, and tiredness can often be felt.
There can be calf enlargement, where calves tend to appear bigger due to fat taking the place of muscular tissue. There can also be cognitive issues. Some people may be intellectually challenged while developing and learning.
People with LGMD have higher levels of a substance called creatine kinase (CK) in their blood. This is a sign that their muscles are damaged. To confirm if someone has LGMD, doctors may need to do a muscle biopsy (a small sample of muscle is tested) and a genetic test to look for changes in the genes that cause this condition.
Here are some of the signs and symptoms to watch for:
- Struggling to get up from the floor
- Having frequent falls
- Walking with a waddling gait
- Complaining of tiredness or muscle cramps
- Needing help climbing stairs or lifting objects
Understanding the overall symptoms as well as clinical features may help healthcare professionals diagnose and manage LGMD more effectively, with competent, targeted, and personalized treatments.
What are the Causes of LGMD?
LGMD is caused by genetic mutations that affect the structure and function of muscles. These mutations prevent muscle cells from working properly, leading to a gradual loss of strength.
Each LGMD subtype is linked to a different gene mutation, which is usually inherited from one or both parents. Based on how these mutations are passed down, LGMD is classified into two main types:
These LGMDs are the result of autosomal dominant inheritance patterns. This implies that the condition can be acquired by inheriting the mutated gene from one of your biological parents.
These LGMDs are the result of autosomal recessive inheritance patterns. This implies that you have inherited a genetic mutation that is responsible for the condition from both of your biological parents.
Can Limb-Girdle Muscular Dystrophy be Inherited?
Limb-Girdle Muscular Dystrophy is an inherited condition. This means it is passed down through families due to changes or mutations in some genes that affect how muscles work.
A child may inherit the faulty gene from one or both parents, depending on the type of LGMD. In many cases, parents may not even know they carry the gene because they don’t have symptoms themselves.
How is Limb-Girdle Muscular Dystrophy diagnosed?
The diagnosis of limb-girdle muscular dystrophy (LGMD) usually involves clinical evaluation, genetic testing, and specific tests.
A physician will perform a thorough physical examination, and while conducting the physical examination, he will look for the presence of muscle weakness and wasting, reduced mobility, and difficulties in performing various day-to-day activities.
It is an important component of the diagnostic process for LGMD. In fact, several different genes cause LGMD, and genetic testing could identify mutations in these specific genes that provoke the disease.
The test may be conducted from a blood sample, saliva sample, or even a muscle biopsy, depending upon the suspected type of LGMD.
Other than genetic testing, specific tests might be applied for assessing the muscle function and the degree of muscle damage.
These can be electromyography, which diagnoses the electrical activity of muscles; nerve conduction studies to test the nerve; and imaging tests like MRI or CT scans.
In general, diagnosis of LGMD is complicated requiring a multidisciplinary approach in the diagnosis with specialized testing and expertise coming from a team of healthcare providers.
Limb-Girdle Muscular Dystrophy Treatment & Management in India
The primary objective of treatment is to improve one’s health and alleviate symptoms. Treatments can vary with the specific type of LGMD and may include:
The primary objective of physical and occupational therapies is to increase muscle strength and flexibility. They can assist you in preserving your mobility and identifying more efficient methods for completing daily duties.
Devices such as canes, supports, walkers, and wheelchairs can boost your mobility and mitigate the risk of falling. Additionally, they can assist in alleviating fatigue.
Using these assistive devices can greatly improve mobility and help with daily tasks. When weakness in the pelvic girdle and upper legs leads to frequent falls, a power wheelchair or scooter can provide a convenient solution.
In order to alleviate tension in contracted muscles and to rectify scoliosis, certain individuals with LGMD may require surgery. For example, surgery may be used to correct scoliosis or other spinal deformities or to release contractures that can occur in the muscles and tendons.
Respirators and cough-assist devices can assist in respiration. Tracheostomy and assisted ventilation may be required in the event of respiratory failure.
The treatment of cardiomyopathy with ACE inhibitors and/or beta-blockers at an early stage may prevent the onset of heart failure and delay the progression of the condition. Pacemakers can also be used to address cardiac rhythm issues and heart failure.
The treatment can thus be supportive to individuals who cannot speak well. People with limb-girdle muscular dystrophy type 1A (LGMD 1A) could also have weakness, particularly in the bulbar muscles, such as the tongue and pharynx. This would easily trigger a problem in speech (dysarthria) and swallowing (dysphagia).
In addition to these conventional treatment options, there are also several emerging therapies and clinical trials being developed to treat LGMD.
These include gene therapy, stem cell therapy, and novel drug therapies that target specific genetic mutations associated with the disease.
Stem cell therapy has the potential to transmit progressive muscle proteins and restore the stem cell pool. This has made it a promising treatment option for muscular dystrophy in India.
Some parents opt for homeopathy as a complementary approach to help manage symptoms of LGMD. Homeopathy may offer mild relief from fatigue, cramps, or emotional stress.
Ayurveda helps improve energy levels, manage muscle fatigue, and support digestion through herbal remedies, massage, and dietary changes.
MedicoExperts’ Combination Therapy for LGMD
If you are looking for a comprehensive approach to managing LGMD for your child, MedicoExperts offers a personalized combination that brings together the goodness of advanced medical science and traditional wisdom.
This approach blends regenerative medicine, Ayurveda, and homeopathy, and it is guided by doctors to target muscle weakness, improve mobility, and support overall health. Each child receives a treatment plan personalized to their age, stage of progression, and specific muscle challenges.
Parents have reported visible progress within weeks: better balance, more energy, and improved physical control. It’s a non-invasive, safe, and structured approach designed to give your child a real chance at independence and a better quality of life.
Want to know whether your child is eligible for our combination therapy? Tell us more about your child’s condition.
How Do We Do It?
Once you connect with MedicoExperts, our team takes the time to understand your child’s case in detail. We collect relevant medical reports or videos and initiate an in-depth discussion with our internal medical board.
This board includes experts across various healthcare sciences who collaboratively design a personalized treatment plan. It involves a carefully sequenced combination therapy approach.
Once the treatment plan is finalized, it is shared with you along with clear reasoning and expected outcomes. Upon your approval and payment, the treatment process begins with full guidance and support from our team.
Takeaway
If your child is diagnosed with LGMD, your first step should be to get the right information. Being informed and proactive makes a world of difference. One of the most promising approaches is MedicoExperts’ combination therapy, which is a holistic treatment model that blends regenerative medicine, Ayurveda, homeopathy, and supportive care to target the condition from multiple angles.
Instead of just managing symptoms, this therapy focuses on boosting muscle strength, energy levels, and growth and overall well-being of your child. Contact us today to give your child an independent, active, and fulfilling life.
Frequently Asked Questions (FAQs):
Q1. How does LGMD affect your daily life?
A: LGMD can make simple activities like walking, and climbing up the stairs or even heights difficult. Affected persons may have to employ the use of assistive devices and modification of the environment so they can remain independent and live successfully with disability.
Q2. What age does limb-girdle muscular dystrophy start?
A: LGMD may appear at any age and generally worsen with time. However, in some cases, they remain mild.
Q3. Is exercise good for muscular dystrophy?
A: Yes, exercises can be beneficial for muscular dystrophy.
Q4. Is combination therapy safe for my child, and when should we consider it?
A: Yes, when supervised by experienced professionals, combination therapy is safe and personalized to your child’s unique needs.
It’s best to consider it early in the diagnosis to preserve muscle function and delay progression. MedicoExperts designs customized plans after a thorough assessment to ensure safe and effective results.
Q5. Can physical activity help relieve LGMD symptoms?
A: Yes, physical activity can help people with LGMD cope with challenges. Physical therapists can create specific exercise programs to help you increase your strength, flexibility, and mobility.
Q6. How can I find an LGMD specialist?
A: Consult your family doctor, contact the Center for Neuromuscular Diseases, or seek out speciality clinics for muscular dystrophy. MedicoExperts can also help you connect with experienced doctors and facilities who can meet your individual needs.
Q7. What role does MedicoExperts play in LGMD treatment?
A: MedicoExperts is crucial in the treatment of LGMD because it bridges one to the best-qualified specialists and advanced treatment facilities. We help in this treatment by helping you find your way in the health care system, ensuring that the treatment is of high quality, and also ensuring cost-effective treatment by by connecting you to the best hospitals and doctors.
Q8. How long can an individual live with limb-girdle muscular dystrophy?
A: The life expectancy for those with LGMD varies greatly depending on the subtype and how rapidly the disease advances.
People who develop heart and respiratory complications tend to have a shorter life expectancy than those who do not have these problems.
Q9. What is the prognosis outlook for those with limb-girdle muscular dystrophy?
A: The prognosis for LGMD is difficult to predict accurately. Healthcare experts will give you as much information as possible about what to expect based on your specific situation.
In general, LGMD that begins in childhood advances quickly and causes more difficulties. In contrast, when LGMD begins in adolescence or adulthood, it is usually slower-progressing and less severe.
Q10. Can limb-girdle muscular dystrophy be prevented?
A: Limb-girdle muscular dystrophy (LGMD) is a genetic disorder, so it is not currently preventable.
Below are the steps to manage limb-girdle muscular dystrophy.
While LGMD itself cannot be prevented, there are ways to treat the condition, delay complications, and improve your quality of life:
- Eat a healthy diet to prevent malnutrition.
- Drink plenty of fluids to avoid dehydration and constipation.
- Get regular light to moderate exercise as recommended by your doctor.
- Maintaining a healthy weight will help to prevent muscular tension.
- Stop smoking to protect your lungs and heart.
- Keep all of your vaccination shots up to date to lower your risk of this condition.
Q11. How does limb-girdle muscular dystrophy progress?
A: According to 2023 research, roughly 63% of those with LGMD endured mobility loss, whereas 34% developed cardiac/respiratory difficulties.
LGMD begins with muscular weakness in the hips and shoulders, progressing to the lower arms, legs, hands, feet, trunk, and head. This may need the use of mobility aids or a wheelchair.
In rare circumstances, LGMD might impair cardiac and respiratory functioning. This potentially leads to heart failure or breathing problems.
Q12. What are the potential risks and complications associated with limb-girdle muscular dystrophy?
A: The challenges of limb-girdle muscular dystrophy (LGMD) vary based on several circumstances.
Various subtypes of LGMD can present with unique complications.
- Timing of Symptoms: The complications can vary based on the age at which symptoms initially manifest.
- Rate of Progression: The rate at which the condition progresses can be a factor in the development of complications.
- Medical care: Availability of full medical care and proper symptom control can be very significant factors in affecting patient outcomes.
Potential complications that could happen include:
- Delays in Development: Difficulty with gross motor function, such as with walking, in cases of LGMD diagnosed at an early age.
- Intellectual and Learning Differences: A person may have problems related to cognitive impairments and learning difficulties.
- Kyphosis/Scoliosis: Spine abnormalities, particularly in cases of childhood-onset LGMD, may comprise both kyphosis and scoliosis or only one of these.
- Restrictive Lung Disease: This can lead to respiratory insufficiency or failure.
- Malnutrition: Difficulties with eating and swallowing lead to nutritional problems.
- https://www.genomicseducation.hee.nhs.uk/genotes/knowledge-hub/limb-girdle-muscular-dystrophy/
- https://www.mountsinai.org/health-library/diseases-conditions/limb-girdle-muscular-dystrophies#:~:text=Outlook%20(Prognosis),reach%20their%20full%20life%20expectancy.
- https://www.mda.org/disease/limb-girdle-muscular-dystrophy#:~:text=The%20shoulder%20girdle%20is%20the,2%20in%20every%20100%2C000%20individuals.
- https://my.clevelandclinic.org/health/diseases/limb-girdle-muscular-dystrophy-lgmd
- https://my.clevelandclinic.org/health/diseases/23538-duchenne-muscular-dystrophy-dmd
- https://www.medicalnewstoday.com/articles/limb-girdle-muscular-dystrophy#summary
- https://journals.lww.com/jcnmd/fulltext/2023/12000/patterns_of_clinical_progression_among_patients.3.aspx
- https://www.mda.org/disease/limb-girdle-muscular-dystrophy/medical-management
- https://www.nhsinform.scot/illnesses-and-conditions/brain-nerves-and-spinal-cord/muscular-dystrophy/limb-girdle-muscular-dystrophy/
- https://www.ncbi.nlm.nih.gov/books/NBK560582/
- https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10652577/
- https://rarediseases.org/rare-diseases/limb-girdle-muscular-dystrophies/
- https://ojrd.biomedcentral.com/articles/10.1186/s13023-023-02897-x
Next in Types of Muscular Dystrophy
Author Bio:
Dr. Khushbu Jain is a dedicated professional with a passion for advancing healthcare through cutting-edge treatments. She has a special interest in researching regenerative medicine and advanced treatment for diseases that are difficult to treat with conventional treatment options. Her deep understanding of these progressive treatments allows her to offer patients personalized and effective solutions for a variety of health concerns.
Content Medically Reviewed By MedicoExperts Editorial & Clinically Review Board
