I know that obtaining deflazacort in the US is quite difficult now. We are waiting for genetic test results for our ambulant (417 meter 6MWD) son.
Based on the 1995 clinical trial resurrected to gain approval for Emflaza the largest improvement in the first twelve weeks was seen amongst those taking prednisone while from week 12 to 52 the largest benefit was seen in those taking deflazacort.
Looking at this Canadian report of clinical trial data on deflazacort treatment from 2012, deflazacort via the 0.9 mg/kg daily Toronto Protocol prolonged ambulation of 76% of 17 participants to belong 15 years of age.
This leaves me wondering if an initial 12 weeks of prednisone treatment followed by deflazacort might produce additional improvement compared to constant use of one or the other. Furthermore I am curious if occasional switching between prednisone and deflazacort might reduce side effects and possibly avoid developing a tolerance to either.
From a realistic perspective fighting between our health insurance provider and Emflaza Cares is likely to delay access to medicine for my son for several months if we initially opt for deflazacort.
I would appreciate the opportunity to read about the experiences of any boys who have switched between prednisone and deflazacort or vice versa.
The FOR DMD study is an evaluation of different dosing schemes including prednisone and deflazacort. It may answer some of these questions when results are released. The study is estimated to complete in October of 2019.
Vamorolone (VBP-15) may become available in perhaps 2020. Hopefully ongoing clinical studies that are available to patients 6 and under will show that Vamorolone has equal or greater efficacy to existing glucocorticoids with reduced side effects. If so I would want to switch my son as soon as Vamorolone is available.
Edasalonexent (CAT-1004) is supposed to announce a phase 3 trial in the second half of 2017. It is said to be well tolerated and that there were improvements in the rate of decline of muscle function across multiple assessments in boys treated with edasalonexent for 12 weeks. This is another potential alternative to glucocorticoids. The parameters for inclusion in the phase 3 trial are unknown at this time.