Manuscripts and Abstracts

FoldRx Manuscripts

Fleming J, Outeiro TF, Slack M, Lindquist SL, Bulawa CE.
Detection of compounds that rescue Rab1-synuclein toxicity. Methods Enzymol. 2008;439:339-51.

FoldRx Abstracts

Suhr OB. THAOS – Transthyretin Amyloidoses Outcomes Survey, a new global, web-based registry. 4th European Conference on Rare Diseases: Patients at the heart of rare disease policy development. ECRD Report. 2007; 246.

Coelho T. Safety and efficacy of orally administered Fx-1006A in patients with familial amyloid polyneuropathy (FAP): a phase II/III, randomized, double-blind, placebo-controlled study. 4th European Conference on Rare Diseases: Patients at the heart of rare disease policy development. ECRD Report. 2007; 246.

Coelho T, Waddington-Cruz M, Plante-Bordeneuve V, et al. Correlation of clinical outcomes and disease burden in patients with transthyretin (TTR) amyloid polyneuropathy: study Fx-005, a landmark clinical trail of Fx-1006A, a novel small molecule TTR stabilizer. J Neurol. 2008; 255(Suppl 1): 78.

Plante-Bordeneuve V, Suhr OB, Coelho T, et al. THAOS – Transthyretin Amyloidoses Outcomes Survey, a new global, web-based registry. J Neurol. 2008; 255(Suppl 1): 78.

Waddington-Cruz M, Coelho T, Maia L, et al. A landmark clinical trial of a novel small molecule transthyretin (TTR) stabilizer, Fx-1006A, in patients with TTR amyloid polyneuropathy: a phase II/III, randomized, double-blind, placebo-controlled study. J Neurol. 2008; 255(Suppl 1): 107.

Coelho T, Maia L, Martins de Silva A, et al. A Landmark Clinical Trial of a Novel Small Molecule Transthyretin Stabiliser, Fx-1006A, in Patients with TTR Amyloid Polyneuropathy: A Phase II/III, Randomised, Double-Blind, Placebo-Controlled Study. VII FAP symposium. 2008. In press.

Planté-Bordeneuve V, Suhr OB, Coelho T. THAOS - Transthyretin Amyloidoses Outcomes Survey, A New Global, Web-Based Registry. VII FAP symposium. 2008. In press.

Falk RH, Packman J, Grogan D. THAOS - Transthyretin Amyloidoses Outcomes Survey, A New Global, Web-Based Registry. 58th annual meeting of the American Society of Human Genetics. 2008.

Fleming J, Bulawa CE, Packman J, et al. Fx-1006A: A Novel Small Molecule Stabilizer of Tetrameric Wild-Type (wt) and Variant Transthyretin (TTR). 58th annual meeting of the American Society of Human Genetics. 2008. 

Ruberg FL, Skinner M, Kim A, et al. Morbidity and Mortality of Transthyretin (TTR) Amyloid Cardiomyopathy (ATTR-CM): a Prospective Evaluation. Circulation. 2008;118:S_717.

Appelbaum E, Harrigan C, Ruberg FL, et al. Increased Left Ventricular Wall Thickness in Transthyretin-Type Cardiac Amyloidosis as Compared to Primary Light-Chain Cardiac Amyloidosis. J Am Heart. 2008. In press.