• Global FKRP Registry: observations in more than 300 patients with Limb Girdle Muscular Dystrophy R9 

      Murphy, Lindsay B.; Schreiber-Katz, Olivia; Rafferty, Karen; Robertson, Agata; Topf, Ana; Willis, Tracey A.; Heidemann, Marcel; Thiele, Simone; Bindoff, Laurence; Laurent, Jean-Pierre; Lochmüller, Hanns; Mathews, Katherine; Mitchell, Claudia; Stevenson, John Herbert; Vissing, John; Woods, Lacey; Walter, Maggie C.; Straub, Volker (Journal article; Peer reviewed, 2020-04)
      Objective The Global FKRP Registry is a database for individuals with conditions caused by mutations in the Fukutin‐Related Protein (FKRP) gene: limb girdle muscular dystrophy R9 (LGMDR9, formerly LGMD2I) and congenital ...
    • Zilucoplan: An Investigational Complement C5 Inhibitor for the Treatment of Acetylcholine Receptor Autoantibody–Positive Generalized Myasthenia Gravis 

      Howard, James F.; Vissing, John; Gilhus, Nils Erik; Leite, Maria Isabel; Utsugisawa, Kimiaki; Duda, Petra W.; Farzaneh-Far, Ramin; Murai, Hiroyuki; Wiendl, Heinz (Journal article; Peer reviewed, 2021)
      Generalized myasthenia gravis (gMG) is an autoimmune disorder in which pathogenic autoantibodies damage the neuromuscular junction, causing disabling or life-threatening muscle weakness. Most treatments nonspecifically ...