Results 21 to 30 of about 270,959 (189)

An insight into bronchiectasis: Causes, clinical features, and treatment practices

Indian Journal of Respiratory Care, 2020
Introduction: Bronchiectasis is a common, progressive respiratory disease characterized by irreversibly dilated, damaged, and thickened bronchi. It is present as a clinical syndrome of chronic cough, sputum production, and recurrent lower respiratory ...
Laxmi Devi, Rajiv Garg, Ankit Kumar, R.A.S. Kushwaha, Santosh Kumar   +4 more
doaj   +1 more source

Classic galactosemia: features of diagnosis and treatment

Zdorovʹe Rebenka, 2018
Background. Galactosemia type I, or classic galactosemia (CG), is the most severe form of hereditary disorders of carbohydrate metabolism, in particular, galactose. Galactosemia is not included in the program of massive neonatal screening in Ukraine. The
N.O. Pichkur, N.V. Olkhovych, Ya.I. Doronina   +2 more
doaj   +1 more source

Pharmaceutical compounding of orphan active ingredients in Belgium : how community and hospital pharmacists can address the needs of patients with rare diseases [PDF]

, 2019
Background: Pharmaceutical compounding of orphan active ingredients can offer cost-effective treatment to patients when no other drug product is available for a rare disease or during periods of drug product shortages.
Boussery, Koen, De Spiegeleer, Bart, Peeters, E, Remon, Jean Paul, Van Tongelen, Inge, Vanhoorne, Valérie, Vervaet, Chris, Wynendaele, Evelien   +7 more
core   +2 more sources

Tracing the Orphan Stream to 55 kpc with RR Lyrae Stars [PDF]

, 2013
We report positions, velocities and metallicities of 50 ab-type RR Lyrae (RRab) stars observed in the vicinity of the Orphan stellar stream. Using about 30 RRab stars classified as being likely members of the Orphan stream, we study the metallicity and ...
Bellm, Eric C., Bhalerao, Varun B., Cohen, Judith G., Grillmair, Carl J., Kulkarni, Shrinivas R., Laher, Russ R., Levitan, David, Ofek, Eran O., Prince, Thomas A., Sesar, Branimir, Surace, Jason A., Tang, Sumin, Waszczak, Adam   +12 more
core   +3 more sources

Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit–The EU Regulator's Perspective

Frontiers in Medicine, 2021
Since the implementation of the EU Orphan Regulation in 2000, the Committee for Orphan Medicinal Products at the European Medicines Agency has been evaluating the benefits of proposed orphan medicines vs. satisfactory treatment methods.
Maria E. Sheean, Frauke Naumann-Winter, Frauke Naumann-Winter, Giuseppe Capovilla, Giuseppe Capovilla, Giuseppe Capovilla, Maria Elisabeth Kalland, Maria Elisabeth Kalland, Eva Malikova, Eva Malikova, Eva Malikova, Segundo Mariz, Darius Matusevicius, Darius Matusevicius, Robert Nistico, Robert Nistico, Brigitte Schwarzer-Daum, Brigitte Schwarzer-Daum, Stelios Tsigkos, Kyriaki Tzogani, Kristina Larsson, Armando Magrelli, Armando Magrelli, Violeta Stoyanova-Beninska, Violeta Stoyanova-Beninska   +24 more
doaj   +1 more source

Deep-Sea Archaea Fix and Share Nitrogen in Methane-Consuming Microbial Consortia [PDF]

, 2009
Nitrogen-fixing (diazotrophic) microorganisms regulate productivity in diverse ecosystems; however, the identities of diazotrophs are unknown in many oceanic environments. Using single-cell–resolution nanometer secondary ion mass spectrometry images of ^(
Dekas, Anne E., Orphan, Victoria J., Poretsky, Rachel S.   +2 more
core   +2 more sources

Remote homology and the functions of metagenomic dark matter

Frontiers in Genetics, 2015
Predicted open reading frames (ORFs) that lack detectable homology to known proteins are termed ORFans. Despite their prevalence in metagenomes, the extent to which ORFans encode real proteins, the degree to which they can be annotated, and their ...
Briallen eLobb, Daniel Aaron Kurtz, Gabriel eMoreno-Hagelsieb, Andrew Charles Doxey   +3 more
doaj   +1 more source

Orphan penumbrae: Submerging horizontal fields

, 2014
We investigate the properties of orphan penumbrae, which are photospheric filamentary structures observed in active regions near polarity inversion lines that resemble the penumbra of regular sunspots but are not connected to any umbra.
Jurcak, J., Rubio, L. R. Bellot, Sobotka, M.   +2 more
core   +1 more source

Drug discovery opportunities at the endothelin B receptor-related orphan G protein-coupled receptors, GPR37 and GPR37L1

Frontiers in Pharmacology, 2015
Orphan G protein-coupled receptors (GPCRs) represent a largely untapped resource for the treatment of a variety of diseases, despite sophisticated advances in drug discovery.
Nicola J Smith, Nicola J Smith
doaj   +1 more source

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

Orphanet Journal of Rare Diseases, 2021
Background Orphan drug designations are a useful proxy to investigate trends in rare disease drug development. Drug developers must receive a designation before they are eligible for the economic incentives of the Orphan Drug Act in the United States. We
Kathleen L. Miller, Lewis J. Fermaglich, Janet Maynard   +2 more
doaj   +1 more source