All Fulcrum Publications

September 23, 2021

Revolutionizing drug discovery in genetically-defined muscle disease using single-cell and high-dimensional datasets

L. Alejandro Rojas, Ayla Ergun, Caroline Sartain, Anthony Accorsi, Richard Lieberman, Anugraha Raman, Joe Maglio, Erin Valentine, Mary Wertz, Elizabeth Townsend, Damon Polioudakis, Ivan Efremov, Lorin A. Thompson, Serena J. Silver, Christopher M. Moxham

September 23, 2021

An Open-Label Study of Losmapimod to Evaluate the Safety, Tolerability, and Biomarker and Clinical Outcome Assessment Changes in Subjects with FSHD1

Joost Kools, Nicol Voermans, KarlienMul, Lucienne Ronco, John Jiang, Jennifer Shoskes, Kelly Marshall, Diego Cadavid, Michelle L. Mellion, Baziel van Engelen

September 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

September 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

September 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

April 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

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

March 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

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