AveX­is of­fers com­pelling rea­sons for a fast FDA OK for gene ther­a­py to treat spinal mus­cu­lar at­ro­phy in in­fants

For most in­fants suf­fer­ing from spinal mus­cu­lar at­ro­phy type 1, the chances are quite high that they could die or re­quire con­stant res­pi­ra­to­ry as­sis­tance ear­ly on to al­low them to keep breath­ing, with no mo­tor func­tion abil­i­ty for sim­ple things like sit­ting unas­sist­ed. Most die be­fore the age of 4.

But in a small Phase I study of 15 pa­tients treat­ed with AveX­is’ $AVXS gene ther­a­py for SMA, all of the pa­tients were able to reach 20 months with­out the need for con­stant res­pi­ra­to­ry sup­port, or dy­ing. And many achieved im­prove­ments in mo­tor symp­toms, speak­ing to the po­ten­tial — though still un­proven — of this as a once-and-done ther­a­py for a rare but dire ail­ment.

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