[1][3] The most common side effects include headache, fever, fall, cough, vomiting, abdominal pain, cold symptoms (nasopharyngitis) and nausea.

[3] As a first-generation medication, golodirsen is far away from being curative; clinical trial outcomes have demonstrated it to have a marginal effect on ameliorating Duchenne muscular dystrophy pathology.

[3] As of December 2019, golodirsen is approved for therapeutic use in the United States, as well as in the countries that automatically recognize the decisions of the US Food and Drug Administration, under the condition that its benefit will be demonstrated in a confirmatory clinical trial.

[citation needed] Also, the accelerated approval of golodirsen has paved the way for people to have early access to the medication, at the same time, it is shrouded with controversy over a number of issues.

[citation needed] Golodirsen was developed by collaborative research led by Prof. Steve Wilton and Prof. Sue Fletcher in the Perron Institute and licensed to Sarepta Therapeutics by the University of Western Australia.

[2] The pharmacological assessment of golodirsen did not include special population groups, e.g., pregnant and lactating women, the elderly, and people with concurrent disease states.