March 18, 2021
Quantitative Muscle Analysis in FSHD Using Whole Body MRI: Composite Muscle Measurements for Cross Sectional Analysis
Per Widholm, Markus Karlsson, André Ahlgren, Olof Dahlqvist Leinhard, Rabi Tawil, Kathryn Wagner, Jeffrey Statland, Leo Wang, Perry Shieh, Baziel Van Engelen, Diego Cadavid, Lucienne Ronco, Adefowope Odueyungbo, Jay Han, Maya Hatch, Michelle L. Mellion
March 18, 2021
In-Home Passive Measurements of Mobility and Sleep in FSHD Patients and their Relationship with Clinical Disease Severity Metrics
Rumen Hristov, Hariharan Rahul, Zachary Kabelac, Vicky Chan, Maya Hatch, Jay Han, Diego Cadavid, Michelle Mellion, Dina Katabi
December 7, 2020
In vitro characterization of FTX-6058, a novel small molecule fetal hemoglobin inducer for sickle cell disease
Billy Stuart, Pete Rahl, Kingsley Appiah, Ivan Efremov, Lorin A Thompson, Owen Wallace, Christopher Moxham
December 7, 2020
FTX-6058, a novel HbF-inducing agent for the treatment of Sickle Cell Disease and b-Thalassemia
Billy Stuart, Keqiang Xie, Mark Roth, Steven Kazmirski, Kinglsey Appiah, Richard Barnes, Qingyi Li, David Peters, Lucienne Ronco, Kim Stickland, Paul Bruno, Diego Cadavid, Lorin Thompson, Owen Wallace, Ivan Efremov, and Peter Rahl
December 7, 2020
In vivo characterization of FTX-6058, a novel small molecule fetal hemoglobin inducer for sickle cell disease
Keqiang Xie, Mark Roth, Ivan Efremov, Serena Silver, Lucienne Ronco, Lorin A Thompson, Kim Stickland, Christopher Moxham and Owen Wallace
October 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
October 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
October 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