Results 11 to 20 of about 109,656 (184)

Orphan drugs

Medicinski pregled, 2013
Introduction. Drugs used for treatment of rare diseases are known worldwide under the term of orphan drugs because pharmaceutical companies have not been interested in ?adopting? them, that is in investing in research, developing and producing these drugs.
Svetlana Golocorbin-Kon, Aleksandra Vojinovic, Mladena Lalic-Popovic, Nebojsa Pavlovic, Momir Mikov   +4 more
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Racial and ethnic disparities in COVID-19 mortality in the United States

Journal of Ideas in Health, 2022
Background: Among COVID-19–associated deaths reported in the United States (U.S.), minority communities were disproportionately represented. The objective was to assess differences in mortality by race and ethnicity among patients with coronavirus ...
Francisco Javier Prado-Galbarro, Ana Estela Gamiño-Arroyo, Copytzy Cruz-Cruz, Juan-Manuel Martínez-Núñez, Carlos Sanchez-Piedra   +4 more
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

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

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

Orphan drugs [PDF]

Nature, 1992
Diseases lacking in satisfactory therapies, named orphans (over 60% of the known), raise the problem of the availability of new drugs to be discovered and evaluated on toxicological and clinical bases. The estimated cost for the full development of a drug makes it not profitable (and therefore orphan drug) in the case of rare diseases and the typical ...
openaire   +4 more sources

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

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

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