Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

In this combined population, a nominally significant improvement from baseline was observed for the cipaglucosidase alfa plus miglustat group for Hex4 and CK levels, manual muscle test (MMT), and 6-min walk distance (6MWD), while a nominally significant worsening from baseline was observed for the alglucosidase alfa plus placebo group for CK levels, Gower’s Maneuver, Stairs, Gait, Chair (GSCG), and forced vital capacity (FVC; Figure 13 ) ( 55 ).

also in 7,732 other papers