Other Drugs and Dietary Supplements

Along with steroids, other drugs approved for use in Duchenne include EXONDYS 51 (eteplirsen), which is approved in the US by the Food and Drug Administration (FDA), and ataluren (Translarna), which is approved for use in several European countries by the European Medicines Agency (EMA) but not in the US. Both EXONDYS 51 (eteplirsen) and ataluren (Translarna) are for use in people with distinct types of Duchenne mutations.

EXONDYS 51 is indiciated for people living with Duchenne who have genetic mutations amenable to exon 51 skipping (about 13 percent of cases). This drug aims to “skip” over the flawed portion (exon) to produce partially functional dystrophin.

Ataluren is indicated for people living with Duchenne who have a non-sense mutation (about 13 percent of cases). Although ataluren’s mechanism of action is unknown, it is thought to interact with the part of the cell that “reads” proteins allowing the cell to “read through” non-sense mutations to produce a functional protein.

There are a number of clinical trials underway in Duchenne that are targeted to specific genetic mutations causing the disease. Genetic testing is important to know whether you might be eligible to participate in these trials. To help clinical trial sites find patients who might be eligible for trials, be sure to register in your national Duchenne patient registry – a list can be found here: http://www.treat-nmd.org/resources/patient-registries/list/DMD-BMD.

In addition to the medications discussed above, there are many other drugs and supplements that may be used in Duchenne, but are not approved by the FDA or EMA. Though some of the drugs mentioned in Box 3 are widely used, there is just not enough evidence to say whether these supplements may be helpful or harmful to you. It is important to discuss all medication with your NMS before you think about adding or stopping medication.

Box 3. Other Drugs and Dietary Supplements Not Approved for Use in Duchenne

The experts considered a range of other drugs and supplements that have been used in some cases for Duchenne treatment. They reviewed published data on these substances to see if there was enough evidence for their safety and efficacy to be able to make recommendations.

The experts concluded the following:

  • The use of oxandrolone, an anabolic steroid, is not recommended
  • The use of Botox is not recommended
  • There was no support for the systematic use of creatine. A randomised controlled trial of creatine in Duchenne did not show a clear benefit. If a person is taking creatine and has evidence of kidney problems, this supplement should be discontinued
  • No recommendations can be made at this time about other supplements or other drugs that are sometimes used in Duchenne treatment, including co-enzyme Q10, carnitine, amino acids (glutamine, arginine), anti-inflammatories/antioxidants (fish oil, vitamin E, green tea extract, pentoxifylline), and others including herbal or botanical extracts. The experts concluded that there was not enough evidence in the published literature to support their use
  • The experts agreed that this is an area where additional research is needed. Active involvement of families in activities that develop further knowledge, such as patient registries and clinical trials, was encouraged