A phase Ib/IIa, open-label, multiple ascending-dose trial of domagrozumab in fukutin-related protein limb-girdle muscular dystrophy.

Doris G LeungAlex E BocchieriShivani AhlawatMichael A JacobsVishwa S ParekhVladimir BravermanKatherine SummertonJennifer MansourNikia StinsonGenila BibatCarl MorrisShannon MarraffinoKathryn R Wagner

Published in: Muscle & nerve (2021)

We conclude that, although domagrozumab was safe in patients in limb-girdle muscular dystrophy type 2I/R9, there was no clear evidence supporting its efficacy in improving muscle strength or function.

Keyphrases

muscular dystrophy
duchenne muscular dystrophy
open label
end stage renal disease
clinical trial
ejection fraction
study protocol
newly diagnosed
phase iii
phase ii
prognostic factors
peritoneal dialysis
randomized controlled trial
squamous cell carcinoma