Results 331 to 340 of about 13,095,395 (361)

Rare advances for rare diseases

The Lancet Neurology, 2017
openaire   +2 more sources

IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases

Nature Reviews Drug Discovery, 2023
Anneliene Hechtelt Jonker, Simon Day, Day Simon   +2 more
exaly  

Undiagnosed and Rare Diseases

Clinics in Perinatology, 2017
Robert M, Kliegman, Brett J, Bordini
openaire   +2 more sources

Rare diseases: challenges and opportunities for research and public health

Nature Reviews Disease Primers
D. Taruscio, W. A. Gahl
semanticscholar   +1 more source

Targeting shared molecular etiologies to accelerate drug development for rare diseases

EMBO Molecular Medicine, 2023
Galliano Zanello, Pj Brooks, Macarena Garrido-Estepa   +2 more
exaly  

Rare Presentation in a Rare Disease

B55. GETTING TO THE HEART OF CARDIOPULMONARY ANOMALIES AND DIAGNOSTIC DILEMMAS IN PH, 2023
A.M. Al-Dobyany, H.A.A. Alsulami, I.S. Khallaf, A. Touman, N. Ghaleb, E. Alqurashi   +5 more
openaire   +1 more source

Children and young people's experiences of living with rare diseases: An integrative review

Journal of Pediatric Nursing, 2023
Philip Larkin
exaly  

[Rare diseases].

Fortschritte der Neurologie Psychiatrie, 2023
Heinz Reichmann
semanticscholar   +1 more source

How many rare diseases are there?

Nature Reviews Drug Discovery, 2020
Nicole Vasilevsky, Nomi Harris, Peter N Robinson   +2 more
exaly  

Rare diseases and orphan drugs

Nature Reviews Drug Discovery, 2012
Irena Melnikova
exaly