by Corticosteroid Treatment
One of the mainstays of current Duchenne muscular dystrophy treatment is corticosteroid medication. Corticosteroids like prednisone and deflazacort work to slow the loss of muscle function and strength in boys with DMD. By decreasing inflammation in the body, corticosteroids can preserve ambulation and delay the progression of the condition by up to 2-5 years. However, long-term corticosteroid use also comes with potential side effects like weight gain, stunting of growth, behavioral changes, and weakened bones that increase the risk of fractures. Doctors carefully monitor patients on corticosteroids and may adjust dosages to balance treatment benefits with quality of life impacts.
Exon Skipping Therapy
A precision medicine approach called exon skipping uses antisense oligonucleotides (ASOs) to manipulate gene splicing and “skip over” specific exons in the dystrophin gene. By inducing the exclusion of particular exons during pre-mRNA splicing, exon skipping can restore the dystrophin reading frame in around 13% of Duchenne patients. The resulting partly functional dystrophin protein has clinical benefits, reducing degradation of muscle fibers. Eteplirsen was the first exon 51 skipping drug approved for Duchenne Muscular Dystrophy Treatment in 2016. Other exon skipping drugs in clinical trials target different exons with the goal of developing personalized treatment regimens. Exon skipping holds promise but comes with a high cost and requires lifelong adherence.
Gene Therapy Clinical Trials
Gene therapy aims to directly deliver a functional copy of the missing dystrophin gene to muscle cells. Viral vectors like adeno-associated viruses are altered to carry the normal dystrophin gene sequence. Injection into muscles allows targeted gene transfer and expression of the protein. Several Duchenne gene therapy candidates have moved into human clinical trials in recent years. An AAVrh74.MHCK7.micro-dystrophin construct tested by Sarepta Therapeutics showed signs of safety and efficacy. Pfizer’s PF-06939926 and Solid Biosciences’ SGT-001 also aim to restore functional dystrophin and halt disease progression through in vivo gene therapy approaches. Success in clinical trials would revolutionize DMD treatment if gene therapy can be scaled up for widespread use.
Assistive Devices
Mobility aids help boys with Duchenne muscular dystrophy preserve independence and quality of life as muscle weakness progresses. Walkers, braces, wheelchairs, and other devices compensate for loss of ambulation. Ankle-foot orthoses (AFO braces) worn in shoes can improve walking capability. Later, boys transition to full-time wheelchair use as leg muscles become unable to support standing and walking. Accessible home modifications, wheelchair vans, and learning skills for self-care from a seated position are important accommodations. Adaptive devices empower individuals to participate fully in education, work, and community activities despite physical limitations.
Multidisciplinary Care
Duchenne muscular dystrophy drastically impacts multiple body systems over time. That’s why comprehensive multidisciplinary care is crucial for optimizing outcomes and functions. A team of specialists coordinates treatment and manages complications under a neuromuscular physician. Physical and occupational therapists provide range-of-motion and strengthening exercises. A respiratory therapist monitors lung function decline preventing infections. A cardiologist monitors for cardiomyopathy. A nutritionist addresses weight management and dietary needs. Mental health professionals help cope with the psychosocial aspects of a chronic condition. Well-coordinated multidisciplinary care is proven to extend ability, independence, and quality of life for individuals with DMD.
Emerging Duchenne Muscular Dystrophy Treatment
Beyond established therapies, researchers actively explore additional emerging Duchenne treatment strategies. Antifibrotics may reduce scarring and fibrosis in muscles. Nonsense suppression drugs seek to induce read-through of premature stop codons in the dystrophin gene caused by certain mutations. Stem cell therapies aim to regenerate muscles through engraftment of healthy stem and progenitor cells. CRISPR/Cas9 genome editing holds potential for directly correcting the underlying genetic defect. Anti-inflammatory and immunomodulatory compounds target specific pathways implicated in DMD pathology. Many new avenues are in preclinical development and testing phases with the goal of ultimately finding a cure through restoring full-length functional dystrophin. Only ongoing research can continue expanding treatment options for Duchenne muscular dystrophy patients.
While no cure exists yet for Duchenne muscular dystrophy, significant progress has been made in recent decades improving life expectancy and quality of life for individuals with this condition. A variety of pharmacological, genetic, and supportive therapies actively work to slow disease progression at different levels. From corticosteroids to exon skipping drugs to emerging gene and stem cell therapies, the medical toolbox for Duchenne Muscular Dystrophy Treatment continues expanding based on understanding disease mechanisms. Success in clinical trials could validate new treatments to benefit future generations. With a multidisciplinary care approach and innovations stemming from dedicated research, many aims to empower independence for as long as possible remain within reach for boys with Duchenne muscular dystrophy.
*Note:
1. Source: Coherent Market Insights, Public Source, Desk Research
2. We have leveraged AI tools to mine information and compile it.
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