Publications & Presentations

Sep 23, 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

Sep 23, 2021

Use of snRNA-seq to characterize the pathogenic skeletal muscle microenvironment

Anugraha Raman, Anthony Accorsi, Michelle Mellion, Bobby Riehle, Lucienne Ronco, L. Alejandro Rojas, Christopher Moxham

Sep 23, 2021

Whole Body MRI Quantitative muscle analysis to evaluate Efficacy of Losmapimod in a Phase 2 Placebo-Controlled Study in Subjects with FSHD (ReDUX4): ReDUX4 (NCT04003974)

Rabi Tawil, Kathryn Wagner, Jeffrey Statland, Leo Wang, Angela Genge, Sabrina Sacconi, Hanns Lochmüller, David Reyes Leiva, Jordi Diaz-Manera, Jorge Alonso-Perez, Nuria Muelas, Juan J. Vilchez, Alan Pestronk, Summer Gibson, Namita A. Goyal, Johanna Hamel, Lawrence Hayward, Nicholas Johnson, Samantha LoRusso, Miriam Freimer, Perry B. Shieh, Sankarasubramoney H. Subramony, Doris Leung, Baziel van Engelen, Joost Kools, Olof Dahlqvist, Per Widholm, John Jiang, Adefowope Odueyungbo, William Tracewell, Alisa Rahilly, Alejandro Rojas, Anthony Accorsi, Steve Mennen, Lucienne Ronco, Diego Cadavid, Christopher M. Moxham, Christopher Morabito, Robert Gould, Michelle L. Mellion

Apr 9, 2021

Discovery of Clinical Candidate FTX 6058: A Potent, Orally Bioavailable Upregulator of Fetal Hemoglobin for Treatment of Sickle Cell Disease

Ivan V. Efremov, Kingsley Kofi Appiah, Angela Cacace, Yanfei Dong, Shawn D. Johnstone, Steven Kazmirski, Qingyi Li, Christopher Moxham, Peter Rahl, Mark Roth, Billy Stuart, Lorin A. Thompson, III, Owen B. Wallace, Keqiang Xie, Feng Zhou

Mar 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

Mar 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

Mar 18, 2021

An Optimized Timed Up and Go (TUG) for the Facioscapulohumeral Dystrophy (FSHD) Community

Vicky Chan, Maya Hatch, Michelle Mellion, Diego Cadavid, Jay Han

Dec 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

Dec 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

Dec 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