As mentioned before, there are very few effective treatments currently for muscular dystrophies, with glucocorticoids being the most effective but only served to delay the onset of symptoms. Given that every cell within a patient's body would have the disease mutations, a cure may still be a far away goal, but new strategies for treatment are being explored, which most of them falling into three categories: gene therapy, cell therapy, and drug therapy⁷.

Gene Therapy
Exon Skipping: Muscular dystrophies are often caused by mutations in genes encoding proteins within a specific protein complex, so one strategy is to modify how the gene mRNA is spliced so the mutation is cut out of the mRNA. Given that you are cutting out part of the code for the protein, the resulting product will not be the same as the unaffected version, but it may be more functional than the patient's version. This is done using fragments of nucleic acids that bind to the target regions of the affected gene, and thus hide the region during splicing. This has been mostly applied in relation to DMD in order to relieve the phenotype to level closer to that of BMD patients. This therapy has recently undergone successful proof of concept testing, showing that exon 51 can be successfully skipped and results in increased dystrophin expression¹³. Although an exciting development, "exon skipping" would still be a lifelong regimen for the patient.

Readthrough: About 15 percent of DMD mutations are premature stop codon point mutations that produce an abnormally short or absent protein. New treatments are being developed that cause misreadings at the premature stop codon, but not at the normal one. These misreadings cause the translation machinery to read through the mutated stop codon, inserting an amino acid in the place of the codon and continuing translation. This produces a relatively normal length protein and, hopefully, a relatively normally functioning protein. This strategy has completed the human safety trials and has moved on to moderately sized human trials to gauge its effectiveness as a treatment⁷.

Gene Transfer: Viruses have also been proposed as a way to get the unaffected gene into the cells by using the viruses as vectors to "infect" the muscle cells and produce normal protein. This therapy has a problem in that the leading vector for human gene therapy has the capacity to hold 5 kb, but the full dystrophin gene is about 11 kb. Researchers have effectively used shortened dystrophin genes in mice, but human safety trials have only recently started, and so its effectiveness in humans is still theoretical¹⁰.

Cell Therapy
Satellite Cells: Satellite cells are the body's natural way of regenerating muscle fibers, so it is logical that researchers would attempt to make use of them to treat muscular dystrophies. Researchers have been able to grow and isolate a pure population of satellite cells, and invection of them has shown promise in animal models, but human trials have not shown the same effectiveness. There is also the problem of not being able to use the patient's own satellite cells as those would have the same defect. This would require anti-rejection treatment so that the patient's immune system does not kill the injected cells¹².

Drug Therapy
Myostatin: Myostatin is a negative regulator of muscle growth. Mutations that result in the loss of myostatin function result in muscle overgrowth, with about a doubling in muscle mass in humans. Researchers have begun to look at the possibility of interfering in the myostatin pathway as a method of therapy. Tests of myostatin-specific antibodies and other interfereing agents have shown promise in animal models, and the antibody has moved on to clinical trials after being proved safe in humans⁷.

Insulin-like Growth Factor-1 (IGF-1): IGF-1 is a growth factor involved in the growth of a number of tissues, muscle being one of them. Overexpression of IGF-1 results in a large increase in muscle mass⁸.  Administration of IGF-1 in mice increased the size of the myofibers, conferred some protection against contraction damage, and an increase in the number of muscle fibers, all of which ease the disease phenotype. Though it shows promise, the wide effect of IGF-1 means that lifelong adminstration of it might have negative effects elsewhere in the body such as on cell or organ size¹⁰.