Developing a cure for Black Bone Disease

por Sireau, Nicolas T
Publicado 2012

Findacure – the Fundamental Diseases Partnership

por Hall, Anthony K, Sireau, Nicolas T
Publicado 2014

A patient survey on the impact of alkaptonuria symptoms as perceived by the patients and their experiences of receiving diagnosis and care

por Rudebeck, Mattias, Scott, Ciarán, Sireau, Nicolas, Ranganath, Lakshminarayan
Publicado 2020

Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium

por Rudebeck, Mattias, Scott, Ciarán, Rhodes, Nicholas P., van Kan, Christa, Olsson, Birgitta, Al-sbou, Mohammed, Hall, Anthony K., Sireau, Nicolas, Ranganath, Lakshminarayan R.
Publicado 2021

Comparing nitisinone 2 mg and 10 mg in the treatment of alkaptonuria—An approach using statistical modelling

por Ranganath, Lakshminarayan R., Milan, Anna M., Hughes, Andrew T., Khedr, Milad, Norman, Brendan P., Alsbou, Mohammed, Imrich, Richard, Gornall, Matthew, Sireau, Nicolas, Gallagher, James A., Jackson, Richard
Publicado 2021

Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases

por del Álamo, Marta, Bührer, Christoph, Fisher, Dirk, Griese, Matthias, Lingor, Paul, Palladini, Giovanni, Sireau, Nicolas, Hivert, Virginie, Sangiorgi, Luca, Guillot, Florence, Halftermeyer, Juliane, Soucková, Lenka, Nosková, Kristýna, Demlová, Regina
Publicado 2022