Results 1 to 10 of about 270,954 (184)
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 +7 more
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Asas wa Tandhim, 2022 This study aims to examine using a structural approach regarding the institutional scope of world bodies in manifesting Acturian Actors in the context of global children's rights as a strengthening of regulations on sustainable custody that must be ...
Agus Pandoman
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Arginase 1 Deficiency: using genetic databases as a tool to establish global prevalence
Orphanet Journal of Rare Diseases, 2022 Background/objective Arginase 1 Deficiency (ARG1-D) is a rare inherited metabolic disease with progressive, devastating neurological manifestations with early mortality and high unmet need.
C. Catsburg +3 more
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Frontiers in Public Health, 2023 Orphans, especially those who experience maternal loss at a young age, face significant long-term negative impacts on their lives and psychological well-being, extending beyond the age of 18. As of July 2023, the global death toll of COVID-19 has reached
Viroj Tangcharoensathien +7 more
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Characterisation of Type II DNA Methyltransferases of Metamycoplasma hominis
Microorganisms, 2023 Bacterial virulence, persistence and defence are affected by epigenetic modifications, including DNA methylation. Solitary DNA methyltransferases modulate a variety of cellular processes and influence bacterial virulence; as part of a restriction ...
Lars Vogelgsang +6 more
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The Astrophysical Journal, 2010 We explore the origin of a population of distant companions (~1000 - 5000 AU) to Class I protostellar sources recently found by Connelley and co-workers, who noted that the companion fraction diminished as the sources evolved. Here we present N-body simulations of unstable triple systems embedded in dense cloud cores.
Reipurth, Bo +3 more
openaire +2 more sources
Molecular Genetics and Metabolism Reports, 2019 Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disorder (LSD) characterized by severe central nervous system (CNS) degeneration.
Susanne Gustavsson +11 more
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Rwanda Medical Journal, 2022 ACKGROUND: Nigeria has the second-highest burden of stunted children globally. Yet, only two out of ten malnourished Nigerian children benefit from intervention programs to address malnutrition.
O. A. T. Fatunla +6 more
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BMC Health Services Research, 2020 Background Classical homocystinuria (HCU), an inborn error of homocysteine metabolism, has previously been estimated to affect approximately 1 in 100,000–200,000 people in the United States (US).
Marcia Sellos-Moura +5 more
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