Results 141 to 150 of about 912,676 (170)

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

Nature Reviews Drug Discovery, 2023
Anneliene H Jonker, Simon Day, Anna Maria Gerdina Pasmooij   +2 more
exaly  

Undiagnosed and Rare Diseases

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

The Cost of Rare Diseases

JAMA, 2021
openaire   +2 more sources

Targeting shared molecular etiologies to accelerate drug development for rare diseases

EMBO Molecular Medicine, 2023
Galliano Zanello, Macarena Garrido-Estepa, Ana Crespo   +2 more
exaly  

Being the Pillar for Children with Rare Diseases—A Systematic Review on Parental Quality of Life

International Journal of Environmental Research and Public Health, 2021
Johannes Boettcher, Michael Boettcher, Silke Wiegand-Grefe   +2 more
exaly  

How many rare diseases are there?

Nature Reviews Drug Discovery, 2020
Melissa A Haendel, Nicole A Vasilevsky, Deepak R Unni   +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  

A systematic review on machine learning approaches in the diagnosis and prognosis of rare genetic diseases

Journal of Biomedical Informatics, 2023
JOSÉ A López-Escámez
exaly  

Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

Nature Reviews Drug Discovery, 2019
Benjamin Vandendriessche, Philip J Brooks, Kristina Larsson   +2 more
exaly