Results from VYONDYS 53 clinical studies.
Researchers conducted clinical trials of VYONDYS 53 to review different aspects of the medicine, including whether it triggers skipping of exon 53 on the dystrophin gene. They also analyzed the amount of dystrophin produced, along with its safety profile. VYONDYS 53 was studied in boys who had a confirmed mutation in the dystrophin gene that could be treated by skipping exon 53. Learn what this means.
Designed to skip exon 53.
One clinical study looked at whether exon skipping occurred on the dystrophin gene of boys treated with VYONDYS 53. In clinical studies, exon skipping occurred in all 25 evaluated study participants.
Dystrophin levels increased in some clinical trial participants.
A study of 25 boys with Duchenne muscular dystrophy compared the level of dystrophin in their bodies before the first infusion (baseline) of VYONDYS 53 with the level of dystrophin after 48 weeks of treatment with VYONDYS 53. Weekly infusions of VYONDYS 53 helped the body make a shorter form of the dystrophin protein. The amount of dystrophin produced varied in the study.
Dystrophin levels measured by western blot can be strongly affected by differences in how the sample is processed, the reference material used, and the quantification method.
Comparing dystrophin results from other methods of measurement requires standard reference material and additional studies.
What do the results tell us?
What about risks? Researchers assessed the risks and tolerability of VYONDYS 53. As with any medication, you should discuss the risks and side effects associated with VYONDYS 53 with your doctor.