Results 31 to 40 of about 3,868,024 (353)

Contribution of psychoactive substance use and other environmental factors to adolescent pregnancies in Mexico

Public Health and Toxicology, 2023
Introduction Mexico has the highest prevalence of adolescent pregnancies among all the member countries of the Organization for Economic Co-operation and Development (OECD) with a fertility rate of 70.6 births for every 1000 women aged 15–19 years.
Francisco-Javier Prado-Galbarro, Carlos Sanchez-Piedra, Juan-Manuel Martinez-Nuñez   +2 more
doaj   +1 more source

Regulatory Standards in Orphan Medicinal Product Designation in the EU

Frontiers in Medicine, 2021
Twenty years of orphan regulation in Europe have now elapsed, with almost 2,400 orphan designated medicinal products and more than 190 orphan products authorised in the EU.
Stelios Tsigkos, Segundo Mariz, Maria Elzbieta Sheean, Kristina Larsson, Armando Magrelli, Armando Magrelli, Violeta Stoyanova-Beninska, Violeta Stoyanova-Beninska   +7 more
doaj   +1 more source

Pediatric Orphan Drug Indications: 2010-2018. [PDF]

Pediatrics, 2020
One-third (136) of FDA-approved orphan indications between 2010 and 2018 were pediatric. Most pediatric orphan indications expanded use of existing drugs; 20 received breakthrough designation.
Kimmel L, Conti RM, Volerman A, Chua KP.
europepmc   +2 more sources

FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development

Orphanet Journal of Rare Diseases, 2020
Background The Office of Orphan Products Development (OOPD) of the United States (U.S.) Food and Drug Administration (FDA) has awarded over 700 grants to conduct clinical trials of medicals products for rare diseases since 1983, leading to over 70 ...
Kathleen L. Miller, Christine Mueller, Gumei Liu, Katherine I. Miller Needleman, Janet Maynard   +4 more
doaj   +1 more source

N-Acetylcysteine Reverses the Mitochondrial Dysfunction Induced by Very Long-Chain Fatty Acids in Murine Oligodendrocyte Model of Adrenoleukodystrophy

Biomedicines, 2021
The accumulation of saturated very long-chain fatty acids (VLCFA, ≥C22:0) due to peroxisomal impairment leads to oxidative stress and neurodegeneration in X-linked adrenoleukodystrophy (ALD).
Jie Zhou, Marcia R. Terluk, Paul J. Orchard, James C. Cloyd, Reena V. Kartha   +4 more
doaj   +1 more source

Molecular Interactions between Gasotransmitters in Patients with Obstructive Sleep Apnea

International Journal of Translational Medicine, 2022
Patients with obstructive sleep apnea (OSA) have an increased risk of cardiovascular disease (CVD). Nitric oxide (NO) and heme oxygenase-1 (HO-1) affect vascular tone and are vasoprotective.
Snigdha Pusalavidyasagar, Laurie B. Hovde, Jessie Lee, Lei Zhang, Adnan Abbasi, Reena V. Kartha   +5 more
doaj   +1 more source

Systematic review of available evidence on 11 high-priced inpatient orphan drugs [PDF]

, 2013
__Background__: Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, costeffectiveness and budget ...
Hakkaart-van Roijen, L. (Leona), Kanters, T.A. (Tim), Redekop, W.K. (Ken), Sonneville, C. (Caroline) de   +3 more
core   +8 more sources

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

Antioxidants, 2023
N-acetylcysteine (NAC), a precursor of cysteine and, thereby, glutathione (GSH), acts as an antioxidant through a variety of mechanisms, including oxidant scavenging, GSH replenishment, antioxidant signaling, etc. Owing to the variety of proposed targets,
Siddhee A. Sahasrabudhe, Marcia R. Terluk, Reena V. Kartha   +2 more
doaj   +1 more source

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

Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021

JAMA Network Open, 2022
This cross-sectional study examines how often and in what combinations 4 US Food and Drug Administration programs for expedited development and review of novel drugs were used among orphan and nonorphan biologics and small-molecule drugs approved between
Andrea N Monge, D. Sigelman, R. Temple, H. Chahal   +3 more
semanticscholar   +1 more source