Santhera and ReveraGen have announced positive topline results for vamorolone, a steroid alternative for individuals living with Duchenne muscular dystrophy (DMD).
Their pivotal VISION-DMD study in boys 4 to <7 years old demonstrated benefits across multiply timed tests compared to placebo (boys not on treatment) and similar benefits to individuals taking prednisone. Vamorolone also showed a favourable safety and tolerability profile, including less of the negative side effects generally associated with steroid treatment, such as delayed growth and weight gain, all significant concerns for families and individuals living with Duchenne.
Santhera will be working with regulatory agencies in the US and Europe to bring vamorolone to Duchenne patients. Defeat Duchenne Canada continues to work with Santhera and ReveraGen to advocate for access in Canada.
Defeat Duchenne Canada awarded Dr. Eric Hoffman and ReveraGen $1 million in 2020:
“Defeat Duchenne Canada has provided the first seed funding of a new trial to expand testing (and hopefully eventual approval) of a broader age range of boys with DMD, including those that have been previously treated with corticosteroids. The clinical trials of vamorolone to date have been only in 4 to <7 years DMD boys never previously treated with prednisone or deflazacort; the Defeat Duchenne Canada funding holds the hope of expanding future access of vamorolone to a much broader group of boys.”Eric Hoffman, CEO of ReveraGen
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“We thank all our Canadian families who have and continue to dedicate their time to participate in these studies. We continue to be very hopeful for what the future holds,” says Director of Research and Advocacy at Defeat Duchenne Canada.Nicola Worsfold
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Director of Research and Advocacy