Pfiz­er gene ther­a­py ups lev­els of dy­s­trophin ex­pres­sion in Duchenne tri­al — but an­oth­er safe­ty set­back mars re­sults

Pfiz­er’s gene ther­a­py group is back up at bat with a fresh up­date of da­ta from its Phase Ib study for a gene ther­a­py in­tend­ed as a once-and-done treat­ment for Duchenne mus­cu­lar dy­s­tro­phy. And they had some good news, and some bad news, to re­port.

The good news was pri­mar­i­ly in the dy­s­trophin ex­pres­sion they tracked among the now 9 evalu­able pa­tients in their first clin­i­cal for­ay for PF-06939926.

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