What is the Best Treatment for Muscular Dystrophy?

Best Treatments for Muscular Dystrophy: Medications and Therapies

Muscular dystrophy (MD) is a group of genetic diseases that cause muscles to weaken and break down over time. There is no cure for any type of MD. Still, a wide range of treatments, including medications, therapies, surgeries, and assistive devices, can significantly lengthen life, improve function, and enhance overall quality of life.

This guide discusses the best techniques for treating muscular dystrophy, new medications being developed, and the most crucial question: Can someone recover from muscular dystrophy? Keep reading for detailed insight.

Medications and Targeted Therapies

a. Corticosteroids

Prednisone and deflazacort are two of the most important drugs for treating Duchenne muscular dystrophy (DMD). These drugs may help keep the muscles strong, slow down scoliosis, improve lung function, and maybe even let us walk on our own for longer. But we need to be very careful with side effects like weight gain, bone thinning, and changes in behaviour.

b. Exon-Skipping Therapies

For DMD patients with certain genetic mutations, antisense medicines help the body "skip" bad gene sequences, which lets the body make a shorter but still useful dystrophin protein. Options that have been approved are:

• Eteplirsen (Exondys 51): For exon 51 mutations
• Golodirsen (Vyondys 53): For exon 53 mutations
• Viltolarsen (Viltepso): Another exon 53 therapy
• Casimersen (Amondys 45): For exon 45 mutations

These medicines slow down the breakdown of muscles, but they aren't a cure and need to be given through an IV every week.

c. Gene Therapy

Elevidys (delandistrogene moxeparvovec), which got the approval in 2023, sends micro-dystrophin through an AAV vector. Early results show increased micro-dystrophin levels in muscle tissue, but the clinical significance of this increase—such as improved mobility or strength—remains under investigation.

d. Novel Medications

Drugs like Ataluren work on premature stop mutations in DMD, and clinical trials show that they work. Givinostat, an HDAC inhibitor, was also approved by the FDA in March 2024 for treatment with all DMD variants. It helps reduce inflammation and muscle atrophy.

Physical, Occupational & Supportive Therapies

There are numerous types of therapy, such as:

• Physical therapy employs customised stretching and low-impact exercise to keep people moving, slow down contractures, and help their breathing muscles. Most programmes involve pool therapy, training in how to move around, and regular checkups every four to six months.
• Occupational therapy helps people stay independent by teaching them how to adapt and suggesting tools that can help. Some types of respiratory therapy use ventilators (such as BiPAP), cough-assist devices, and support for the diaphragm.
• Speech therapy can help with problems with swallowing and speaking, and dietary support and psychological counselling are important parts of overall care.

Surgical and Assistive Interventions

If experiencing severe scoliosis, contractures, eating problems (like gastrostomy), or heart problems (such as pacemakers or defibrillators), surgery is needed. Mobility devices, such as braces, walkers, or electric wheelchairs, are commonly recommended early on to help people maintain their function and independence.

Monitoring and Managing Complications

It is essential to check the health of your heart and lungs regularly:

• ACE inhibitors and beta blockers are heart drugs that slow down heart failure in DMD.
• As the chest muscles get weaker, breathing support becomes crucial.
• Emerging and Investigational Treatments

Some of the most advanced research includes:

• CRISPR gene editing looks promising in early experiments on animalsbut its safety and efficacy in humans remain under investigation, with no approved therapies currently available.
• Vamorolone is a steroid alternative that is being tested in clinical trials.
• Trials for RNA-targeted and anti-inflammatory drugs, such as Avidity's del-brax, which targets FSHD.

These treatments are still being studied and aren't widely available yet.

Can You Fully Recover from Muscular Dystrophy?

No, full recovery is not achievable right now because MD is caused by persistent genetic changes. But:

• Early therapies can greatly slow down the progression of the disease.
• Supportive treatment can help keep people moving and functioning (for example, DMD patients can walk into their late teens with steroids and therapy).
• People with milder types of the disease, such as Becker, LGMD, or FSHD, may live well into adulthood with very little handicap.
• Gene and exon-skipping therapies don't cure illnesses; they just keep them stable.
• Stem cell and CRISPR techniques show promise for the future, although they are still being studied.

Outlook and Longevity by Type

This includes:

• Duchenne MD: Early use of steroids, non-invasive ventilation, and heart care has all helped people live longer, until the mid-30s.
• Becker MD: Less severe symptoms can start at different times; many people live into their 40s or 50s.
• Myotonic MD: Starts in adults and progresses slowly, with almost normal lifespans.
• Limb-Girdle and FSHD: Can be mild to severe, and how long you live depends on the type and quality of care you get.

A multimodal therapy approach that includes drugs, therapies, procedures, and support can reduce the disease's course, maintain strength, and improve quality of life, even though full recovery from muscular dystrophy is not possible. New gene therapies provide us some hope for improvement, but not for reversal.

Families and patients should work with multidisciplinary care teams to change treatment regimens as needed and look into interesting research paths. The future looks better than ever for MD as progress continues, especially in genetic medicines.