Results 281 to 290 of about 4,553,133 (313)

Drug Repurposing for Rare Diseases

Trends in Pharmacological Sciences, 2021
Mieke van Haelst, Gijs van Haaften, Helen Roessler   +2 more
exaly  

Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives

Nucleic Acid Therapeutics, 2022
Matthis Synofzik, Willeke M C van Roon-Mom, Georg Marckmann   +2 more
exaly  

Knowledge-based approaches to drug discovery for rare diseases

Drug Discovery Today, 2022
Anthony Hickey, Sean Ekins, Vinicius Alves   +2 more
exaly  

Rare diseases in ophthalmology

Archivos de la Sociedad Española de Oftalmología (English Edition), 2022
J, Jiménez Benito, M A, Saornil Álvarez, L, Macías Molinero, N, Gajate Paniagua   +3 more
openaire   +2 more sources

N-acetylcysteine Pharmacology and Applications in Rare Diseases—Repurposing an Old Antioxidant

Antioxidants, 2023
Rv Kartha, Siddhee Sahasrabudhe, Marcia R Terluk   +2 more
exaly  

Rare advances for rare diseases

The Lancet Neurology, 2017
openaire   +2 more sources

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

The RareDis corpus: A corpus annotated with rare diseases, their signs and symptoms

Journal of Biomedical Informatics, 2022
Esteban Chacón-Solano, Isabel Segura Bedmar, Guerrero-Aspizua Sara   +2 more
exaly  

New Diagnostic Approaches for Undiagnosed Rare Genetic Diseases

Annual Review of Genomics and Human Genetics, 2020
Taila Hartley, Gabrielle Lemire, Kym Boycott   +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, Holger Zapf   +2 more
exaly