Muscular dystrophy is considered one disease that presents itself in a classic way. While progressive muscle weakness and difficulty controlling movement are present in all nine forms of the disease, there are nuances in the symptoms experienced and their extent that depend on what type someone has.
This disease is mainly caused due to the lack of dystrophin protein. This is primarily a genetic defect hereditary to the parents. Males are more likely to suffer from muscular dystrophy syndrome.
Common Signs and Symptoms of Muscular Dystrophy
Children with muscular dystrophy usually have problems moving around at a young age. They may not sit up, roll over, crawl, or walk as early as other children. These movements are directly affected because muscular dystrophy begins in the shoulders and pelvic area.
In older children and adults, there may be a gradual onset of the following symptoms:
- Clumsiness, tripping and falling.
- Difficulty walking uphill or climbing stairs.
- Inability to jump or hop.
- Toe walking.
- Pain in the pelvis or legs.
- Weakness in the upper body, including shoulders.
- As the disease progresses, additional problems can develop because of muscle weakness. You may experience heart and lung problems.’
Muscular Dystrophy Causes
There are various causes of muscular dystrophy, as follows:
- Gene mutations that occur impulsively
- Family inheritance of the condition
- Due to intimate relation-linked disorder.
- Gender and Age-based developments
- Environmental Factors
- Protein Deficiency
The causes mentioned above are only the significant ones among the known reasons. Due to this disease still being studied, it is possible that more significant causes can be discovered in the future.
Risk Factors and Complications of Muscular Dystrophy
This condition occurs in both sexes and all ages. However, Duchenne’s most common variety usually occurs in young boys. People with a family history of muscular dystrophy are at higher risk of developing the disease or passing it on to their children.
The complications of progressive muscle weakness consist of the following:
- Trouble walking and using arms
- Breathing issues
- Curved spine
- Heart problems
- Swallowing problems
- Shortening of muscles or tendons around joints
What Happens If Muscular Dystrophy Is Not Treated?
Individuals diagnosed with muscular dystrophy characteristically display progressive muscle weakness. For instance, patients with Duchenne muscular dystrophy notice the first symptoms in early childhood.
Initial symptoms include leg weakness, increasing convex curvature of the spine, and a waddle-like gait. Continuous loss of muscle fibers results in muscles that weaken over time, usually forcing DMD individuals to use a wheelchair by age 11 or 12.
In further stages, most patients succumb to cardiac or respiratory failure in their twenties, although rare cases of survival into the thirties have been reported. A similar yet milder type of muscular dystrophy known as Becker muscular dystrophy, along with other types of muscular dystrophy
Treatments Available for Muscular Dystrophy
Conventional treatment includes supportive care that partially reduces signs and symptoms. Such therapies, however, do not reverse the phenotype, nor can they directly target the cause of the pathology. Few treatment options are available for MD, including pharmacological, gene and cell therapy.
- Pharmacological: Various compounds such as allopurinol, vitamin E and selenium, and mazindol, a growth hormone inhibitor, were deemed ineffective or not beneficial against the progression of DMD.
- Gene therapy provides a promising option in treating MD. Still, it requires significant advances in the knowledge of genes, muscle promoters, viral vectors, immune system surveillance and methods for systemic delivery of vectors.
- Stem cell therapy is based on the delivery of autologous and allogeneic cells that contribute to the regeneration of muscle fibers and tissue repair. It is considered a safe and effective option compared to gene and pharmacological therapy. However, it is essential to note that gains in muscle mass and improvements can only be temporary, as stem cell therapy can not treat the defective genes that cause Muscular Dystrophy.
The stem cell treatment in India for muscular dystrophy is followed by proper rehabilitation. It helps improve the patient’s overall potential and simplify their everyday lives. The rehab includes physiotherapy to make the stem cells more functional. Physical and occupational therapies are used to help them in performing daily activities involving the hands and arms.