Sarepta sees muscle function gains with DMD gene therapy
Questex LLC | October 04, 2018
Sarepta’s case for its Duchenne muscular dystrophy (DMD) gene therapy is getting stronger, with early data now linking its effects on a critical protein with improved muscle function. An earlier readout from the phase 1/2 trial in June from three patients treated with the gene therapy showed that it increased levels of microdystrophin a truncated version of the dystrophin protein which is missing in DMD and a fourth patient has now shown “robust” expression levels, says the company.