clinical trial readiness

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1
Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach.
Adang, Laura Ann;Sevagamoorthy, Anjana;Sherbini, Omar;Fraser, Jamie L;Bonkowsky, Joshua L;Gavazzi, F...
2024 Molecular genetics and metabolism DOI: 10.1016/j.ymgme.2024... 48 views
2
Research Letter: The state of academic psychiatry in Aotearoa New Zealand and Australia: Data from a survey of the Royal Australian and New Zealand College of Psychiatrists membership.
Northwood, Korinne;Eggleston, Katherine;Every-Palmer, Susanna;Galbally, Megan;Warren, Nicola;Berk, M...
2024 The Australian and New Zealand journal of psychiat... DOI: 10.1177/000486742412... 48 views
3
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Mercuri, Eugenio;Vilchez, Juan J;Boespflug-Tanguy, Odile;Zaidman, Craig M;Mah, Jean K;Goemans, Natha...
2024 The Lancet. Neurology DOI: 10.1016/S1474-4422(2... 60 views

Related Journals

The Australian and New Zealand journal of psychiatry
ISSN: 0004-8674 686 views
Molecular genetics and metabolism
ISSN: 1096-7206 425 views
The Lancet. Neurology
ISSN: 1474-4465 286 views

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rare disease real world data longitudinal disease trajectory natural history studies novel approach