13 August 2020


VILTEPSO (Vitolarsen) injection has been approved by the FDA for US patients, for the treatment of Duchenne Muscular Dystrophy (DMD) patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping therapy.


NS Pharma advised that VILTEPSO is the first and only exon 53 skipping therapy to demonstrate an increase in dystrophin in children as young as four years old, and that all patients taking VILTEPSO observed an increase in dystrophin production in skeletal muscle levels after treatment.


The most common side effects of VILTEPSO included respiratory tract infection, injection site reaction, cough and fever, with kidney toxicity a possibility.


To learn more:  Viltepso_Approval_Press_Release


“Save Our Sons are vigorously and very regularly advocating to government, the bureaucracy, and key stakeholders, including relevant pharmaceutical companies, on behalf of our Duchenne and Becker community, particularly towards achieving key goals such as securing more places for Australians on clinical trials, and importantly securing more clinical trials in Australia and thus even more places for Australians on clinical trials here at home.


We are making inroads in our quest. We have the attention of key decision-makers and influencers, we are in open and serious ongoing dialogue with them, and we are working very hard to achieve and secure meaningful outcomes for our community.


In terms of our work and efforts, we will make relevant announcements when we can as appropriate and in collaboration with the relevant parties we are advocating to and working with, but please rest assured that we always put our community and their needs first.


Issues pertaining to clinical trials are high on our agenda and are a key objective for us. We will keep you informed on this critically important topic.


Save Our Sons next series of meetings with Commonwealth decision-makers, both elected and bureaucratic, take place in Canberra on 1st September and coincide with the official launch of our Keynote Report which covers all our important issues and objectives, which include getting treatment earlier, getting the care that is needed, funding future and emergent therapies, improving access to clinical trials, and streamlining the domestic approval processes for medications and treatments that have been approved in other jurisdictions such as the USA or in Europe.”