Publications & Presentations

Oct 1, 2020

Development and Evaluation of a Whole-body MRI Protocol and Analysis Algorithms to Measure Changes in Skeletal Muscle in FSHD

Diego Cadavid, Michelle Mellion, Shane Raines, Per Widholm, and Olof Dahlqvist Leinhard

Oct 1, 2020

A Biomarker of Aberrant DUX4 Activity to Evaluate Losmapimod Treatment Effect in FSHD Phase 2 Trials

Lucienne Ronco, Kathryn Wagner, Diego Cadavid, Jayashree Chadchankar, Aaron Chang, Michelle Mellion, Adefowope Odueyungbo, Shane Raines, Anugraha Raman, Alan Robertson, Alejandro Rojas, Ning Shen, Perry Shieh, Jeffrey Statland, Rabi Tawil, Stephen Tapscott, Baziel van Engelen, Leo Wang, Nick Johnson, Owen Wallace

Oct 1, 2020

Open-Label Pilot Study of Losmapimod in FSHD1 (NCT04004000)

Joost Kools, Nicol Voermans, Karlien Mul, Lucienne Ronco, Adefowope Odueyungbo, Venkateswarlu Kondragunta, Kelly Marshall, Mary Bylo, Michelle Mellion, Baziel van Engelen, Diego Cadavid

Oct 1, 2020

A Phase 2, Randomized, Placebo-Controlled, 48-Weeks Study of the Efficacy and Safety of Losmapimod in Treating Subjects with FSHD: ReDUX4 (NCT04003974)

Rabi Tawil, Kathryn Wagner, Jeffrey Statland, Leo Wang, Angela Genge , Hanns Lochmüller, Summer Gibson, Namita Goyal, Johanna Hamel, Lawrence Hayward, Nicholas Johnson, Samantha LoRusso, Jordi Díaz-Manera, Juan J Vilchez- Padilla, Alan Pestronk, Sabrina Sacconi, Perry B. Shieh, S.H.Subramony, Michelle Hage, Adefowope Odueyungbo, William Tracewell, Alisa Rahilly, L. Alejandro Rojas, Michelle Mellion, Lucienne Ronco, Diego Cadavid

May 26, 2020

p38ɑ Regulates Expression of DUX4 in a Model of Facioscapulohumeral Muscular Dystrophy

L. Alejandro Rojas, Erin Valentine, Anthony Accorsi, Joseph Maglio, Ning Shen, Alan Robertson, Steven Kazmirski, Peter Rahl, Rabi Tawil, Diego Cadavid, Lorin A. Thompson, Lucienne Ronco, Aaron N. Chang, Angela M. Cacace, Owen Wallace

Oct 30, 2019

Design of a biomarker of DUX4 activity to evaluate losmapimod treatment effect in FSHD Phase 2 trials

Lucienne Ronco, Diego Cadavid, Jayashree Chadchankar, Aaron Chang, Michelle Mellion, Alan Robertson, Alejandro Rojas, Ning Shen, Rabi Tawil, Stephen Tapscott, Leo Wang, Owen Wallace

Oct 16, 2019

Phase 1 clinical trial of losmapimodin FSHD: safety, tolerability and target engagement

Michelle L. Mellion, Lucienne Ronco, Drew Thompson, Michelle Hage, Sander Brooks, Emilie van Brummelen, Lisa Pagan, Umesh Badrising, William Tracewell, Shane Raines, Baziel van Engelen, Geert Jan Groeneveld, Diego Cadavid

Oct 4, 2019

Safety and tolerability of losmapimod, a selective p38a/b MAPK inhibitor, for treatment of FSHD at its root cause

Diego Cadavid, Michelle Mellion, Owen Wallace, Lucienne Ronco, Drew Thompson, Alejandro Rojas, Michelle Hage, Robert Gould

Oct 4, 2019

Targeting DUX4, the Root Cause of FSHD: Identification of a Drug Target and Development Candidate

Owen Wallace, Anthony Accorsi, Richard Barnes, Angela Cacace, Diego Cadavid, Aaron Chang, Robert Gould, Steven Kazmirski, Joseph Maglio, Michelle Mellion, Peter Rahl, Alan Robertson, Alejandro Rojas, Lucienne Ronco, Ning Shen, Lorin A. Thompson and Erin Valentine, Fulcrum Therapeutics.

Oct 1, 2019

Muscle Xenografts Reproduce Key Molecular Features of Facioscapulohumeral Muscular Dystrophy

Amber L. Mueller, Andrea O'Neill, Takako I. Jones, Anna Llach, Luis Alejandro Rojas, Paraskevi Sakellariou, Guido Stadler, Woodring E. Wright, David Eyerman, Peter L. Jones, Robert J. Bloch