C. Turner

Improving the informed consent process in international collaborative rare disease research: effective consent for effective research

D. Taruscio, Hanns Lochmüller, O. Riess, 2016, European Journal of Human Genetics.

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)

A. De Luca, M. Grounds, K. Nagaraju, 2020, Disease Models & Mechanisms.

Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead

F. Muntoni, E. Heslop, V. Straub, 2020, Neuromuscular Disorders.

Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30–31 January 2017

E. Mazzone, A. Mayhew, E. Mercuri, 2018, Neuromuscular Disorders.

Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA

H. Morizono, R. Finkel, Y. Hathout, 2017, Neuromuscular Disorders.

The Human Phenotype Ontology in 2017

Tudor Groza, Damian Smedley, Peter N. Robinson, 2016, Nucleic Acids Res..

The impact of integrated omics technologies for patients with rare diseases

Hanns Lochmüller, Rachel Thompson, K. Bushby, 2014 .

A decade of optimizing drug development for rare neuromuscular disorders through TACT

A. Mayhew, M. Grounds, N. Goemans, 2019, Nature Reviews Drug Discovery.