دورية أكاديمية

The Arduous Path to Drug Approval for the Management of Prader–Willi Syndrome: A Historical Perspective and Call to Action.

التفاصيل البيبلوغرافية
العنوان: The Arduous Path to Drug Approval for the Management of Prader–Willi Syndrome: A Historical Perspective and Call to Action.
المؤلفون: Singh, Deepan1 (AUTHOR) desingh@maimonidesmed.org, Miller, Jennifer L.2 (AUTHOR) millejl@peds.ufl.edu, Wassman, Edward Robert3 (AUTHOR) drbobwassman@gmail.com, Butler, Merlin G.4 (AUTHOR) mbutler4@kumc.edu, Foley Shenk, Allison3 (AUTHOR) alifoleyshenk.trend@gmail.com, Converse, Monica3 (AUTHOR) maria@trend.community, Picone, Maria3 (AUTHOR)
المصدر: International Journal of Molecular Sciences. Jul2023, Vol. 24 Issue 14, p11574. 3p.
مصطلحات موضوعية: *PRADER-Willi syndrome, *DRUG approval, *MEDICAL genetics, *HUMAN growth hormone, *SELF-injurious behavior
مستخلص: Despite the well-established burden of illness and the social costs of PWS, the only medication that has been approved by the Food and Drug Administration (FDA) for the management of this disorder is recombinant human growth hormone (rhGH), which was approved in 2000 [[7]]. The Arduous Path to Drug Approval for the Management of Prader-Willi Syndrome: A Historical Perspective and Call to Action Prader-Willi syndrome (PWS) is a neuroendocrine genetic disorder resulting from the loss of paternally expressed imprinted genes in chromosome 15q11-q13 [[1]]. [Extracted from the article]
قاعدة البيانات: Academic Search Index
الوصف
تدمد:16616596
DOI:10.3390/ijms241411574