The Arduous Path to Drug Approval for the Management of Prader–Willi Syndrome: A Historical Perspective and Call to Action-Reference-Cited by-同舟云学术

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

Published:2023-07-18 Issue:14 Volume:24 Page:11574
ISSN:1422-0067
Container-title:International Journal of Molecular Sciences
language:en
Short-container-title:IJMS

Author:

Singh Deepan¹,Miller Jennifer L.²,Wassman Edward Robert³^ORCID,Butler Merlin G.⁴^ORCID,Foley Shenk Allison³,Converse Monica³^ORCID,Picone Maria³^ORCID

Affiliation:

1. Department of Psychiatry, Maimonides Medical Center, Brooklyn, New York, NY 11219, USA

2. Department of Pediatrics, Division of Pediatric Endocrinology, University of Florida, Gainesville, FL 32611, USA

3. TREND Community, Philadelphia, PA 19103, USA

4. Departments of Psychiatry & Behavioral Sciences and Pediatrics, University of Kansas Medical Center, Kansas City, KS 66160, USA

Abstract

Prader–Willi syndrome (PWS) is a neuroendocrine genetic disorder resulting from the loss of paternally expressed imprinted genes in chromosome 15q11-q13 [...]

Publisher

MDPI AG

Subject

Inorganic Chemistry,Organic Chemistry,Physical and Theoretical Chemistry,Computer Science Applications,Spectroscopy,Molecular Biology,General Medicine,Catalysis

Link

https://www.mdpi.com/1422-0067/24/14/11574/pdf

Reference13 articles.

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2. Molecular genetic classification in Prader-Willi syndrome: A multisite cohort study;Butler;J. Med. Genet.,2019

3. Prader-Willi syndrome;Cassidy;Genet. Med.,2012

4. Prader-Willi syndrome—Clinical genetics, diagnosis and treatment approaches: An update;Butler;Curr. Pediatr. Rev.,2019

5. Evaluating sleep disturbances in children with rare genetic neurodevelopmental syndromes;Veatch;Pediatr. Neurol.,2021