Randomized, double-blind, placebo-controlled study of interferon-γ 1b in Friedreich Ataxia.

David R LynchLauren HauserAshley McCormickMcKenzie WellsYi Na DongShana E McCormackKim SchadtSusan PerlmanSub H SubramonyKatherine D MathewsAlicia BrochtJulie BallRenee PerdokAmy GrahnTom VescioJeffrey W ShermanJennifer M Farmer

Published in: Annals of clinical and translational neurology (2019)

This study provides no direct evidence for a beneficial effect of IFN-γ1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.

Keyphrases

double blind
placebo controlled
clinical trial
phase iii
dendritic cells
phase ii
immune response
open label
study protocol
minimally invasive
electronic health record
big data
early onset
machine learning
deep learning
artificial intelligence