We will provide an update on the scientific rationale for use of vamorolone, and share some results from our open-label Long Term Extension study. The very important pivotal clinical trial is now seeking participants; we will review information about this trial. ReveraGen was recently awarded a grant to study family and physician preferences regarding the return of individual and aggregate clinical trial data to participant families. Finally, we will discuss ReveraGen’s unique funding model.
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REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial
Sarepta Therapeutics Announces Positive Data for Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
PTC Therapeutics – EMFLAZA® LANDSCAPE IN 2024
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