Muscular dystrophy includes a wide range of debilitating muscle-wasting conditions caused by mutations in genes that are important for muscle maintenance, structure, and function.
Although there is no cure for any type of muscular dystrophy, some approved treatments have been approved for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). Other treatments are also being researched for the treatment of these and other types of muscular dystrophy.
Corticosteroids are medications that resemble an anti-inflammatory hormone produced by the body. They can be prescribed to people with DMD and BMD. Research seems to indicate that reducing inflammation can slow the progression of both types of muscular dystrophy, possibly by preventing inflammation in muscle tissue that might cause additional muscle damage.
Prednisone is an example of a corticosteroid that is prescribed to treat DMD and BMD. Like many corticosteroids, the long-term side effects of prednisone (such as weakening bones and weight gain, among others) can outweigh its benefits.
Emflaza (deflazacort) is a pro-corticosteroid, which can be used to treat DMD symptoms in patients who are 5 or older. Approved by the U.S. Food and Drug Administration (FDA) in February 2017, Emflaza is a prodrug, meaning that the medication has to be metabolized by the body to function as a therapy.
How the therapy works in DMD is not well understood, but scientists know that it has an anti-inflammatory effect, reducing swelling and the damage caused by the immune system attacking healthy tissue.
Several severe side effects can prevent the long-term use of Emflaza.
Exondys 51 (eteplirsen) was the first DMD therapy to receive FDA approval in September 2016. Unlike corticosteroids that target symptoms, Exondys 51 addresses the underlying cause of DMD. The conditional approval covers DMD patients whose disease is caused by specific mutations, potentially being useful to about 13% of all DMD patients. The therapy has not been approved in Europe.
Exondys 51 helps cells produce more dystrophin protein (the protein lacking in DMD) by driving the protein-making machinery of the cell to skip over the mutation.
The treatment is being tested in clinical trials to evaluate its long-term effects and establish its optimal dose in DMD patients.
Translarna (ataluren) is another medication that addresses the underlying cause of DMD and BMD. It is designed to treat DMD and BMD caused by a DMD gene mutation that produces a premature stop signal. This stop signal tells the protein-making machinery of the cell to stop making dystrophin protein before it should. Translarna helps the cellular machinery to skip over the stop signal and produce functional dystrophin protein.
European regulators have approved Translarna, but the treatment has yet to receive U.S. approval.
Translarna’s long-term safety and effectiveness are still being investigated in clinical trials.
Last updated: 07/11/2019
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