The effect of adding real-world evidence to regulatory submissions on the breadth of population indicated for rare disease medicine treatment by the European Medicines Agency

ανά Ravi Jandhyala
Έκδοση 2022-05-01

FGF23 and its role in X-linked hypophosphatemia-related morbidity

ανά Signe Sparre Beck-Nielsen, Zulf Mughal, Dieter Haffner, Ola Nilsson, Elena Levtchenko, Gema Ariceta, Carmen de Lucas Collantes, Dirk Schnabel, Ravi Jandhyala, Outi Mäkitie
Έκδοση 2019-02-01

The international X-linked hypophosphataemia (XLH) registry (NCT03193476): rationale for and description of an international, observational study

ανά Raja Padidela, Ola Nilsson, Outi Makitie, Signe Beck-Nielsen, Gema Ariceta, Dirk Schnabel, Maria Luisa Brandi, Annemieke Boot, Elena Levtchenko, Michael Smyth, Ravi Jandhyala, Zulf Mughal
Έκδοση 2020-06-01