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Catabasis is enrolling boys ages 4 to 7 (up to 8th birthday), any mutation type, who have not been on steroids for at least the past six months.   What is Edasalonexent? Edasalonexent [...]

Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced positive results from its interim analysis of muscle biopsy endpoints comparing casimersen [...]

February 25, 2019 Santhera Pharmaceuticals announces results from the SYROS study.   The primary objective of this study was to evaluate the long-term evolution of the respiratory function [...]