Given that muscular dystrophies occur due to inherited genetic defects in the genes responsible for proper muscle development and functioning, its treatment options are limited, similar to lots of other inherited disorders. Current treatment options for muscular dystrophies include:

• Physiotherapy: Physiotherapy is still the main form of therapy for muscular dystrophy – along with steroids (2).
• Steroids: Steroids are strong anti-inflammatory drugs that can also modulate the body’s immune response. There is current evidence that early use of glucocorticoids/steroids delays the onset of wheelchair dependence. However, to this day and despite extensive studies on the topic, there are still uncertainties regarding which steroid is the best to use and the possible risk of under or over-treatment. This is also in addition to the potential side effects of prolonged steroid use such as weight gain and higher risk of infections (2).
• Gene Therapy: this is yet again another promising mode of therapy – similar to stem cell therapy – with multiple drugs being approved by the FDA for muscular dystrophies due to the limited amount of options available. These drugs target the gene abnormality responsible for muscular destruction and dystrophy; however given that these medications target a certain genetic mutation, they depend on a certain genetic mutation/aberration being present in the patient in order to be able to use it. For example, one drug approved known as Eteplirsen has been reported to be efficacious only in around 15% of patients with DMD who carry a certain mutation in Exon 15 of the DMD gene. This means that in children who have a gene abnormality elsewhere or even a different abnormality within the same gene – which is common – don’t benefit from this medication. Therefore, gene therapy requires a meticulous genetic assessment before its use (3, 4).