2 Works

Data from: Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

Diane E. Frank, Frederick J. Schnell, Cody Akana, Saleh H. El-Husayni, Cody A. Desjardins, Jennifer Morgan, Jay S. Charleston, Valentina Sardone, Joana Domingos, George Dickson, Volker Straub, Michela Guglieri, Eugenio Mercuri, Laurent Servais & Francesco Muntoni
Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Methods Part 1 was a randomized, double-blind, placebo-controlled, 12-week dose titration of once-weekly golodirsen; Part 2 is an ongoing, open-label evaluation. Safety and pharmacokinetics were primary and secondary objectives of Part 1. Primary biological outcome measures of part 2 were blinded exon skipping and dystrophin protein production on muscle biopsies (baseline,...

Supplemental Materials: Identification of proteins associated with the early restoration of insulin sensitivity after biliopancreatic diversion

Cecilia Karlsson, Kristina Wallenius, Anna Walentinsson, Peter Greasley, Tasso Miliotis, Mårten Hammar, Amerigo Iaconelli, Sofia Tapani, Marco Raffaelli, Geltrude Mingrone & Björn Carlsson
ABSTRACT CONTEXT: Insulin resistance (IR) is a risk factor for type 2 diabetes, diabetic kidney disease, cardiovascular disease and non-alcoholic steatohepatitis. Biliopancreatic diversion (BPD) is the most effective form of bariatric surgery for improving insulin sensitivity. OBJECTIVE: To identify plasma proteins correlating with the early restoration of insulin sensitivity after BPD. DESIGN: Prospective single centre study including twenty insulin resistant men with morbid obesity scheduled for BPD. Patient characteristics and blood samples were repeatedly collected...

Registration Year

  • 2020
    2

Resource Types

  • Dataset
    2

Affiliations

  • Agostino Gemelli University Polyclinic
    2
  • Hôpital Armand Trousseau
    1
  • Newcastle University
    1
  • University College London
    1
  • University of London
    1
  • AstraZeneca (Sweden)
    1
  • Sarepta Therapeutics (United States)
    1