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

Deepan Singh; Jennifer L Miller; Edward Robert Wassman; Merlin G Butler; Allison Foley Shenk; Monica Converse; Maria Picone

doi:10.3390/ijms241411574

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

Int J Mol Sci. 2023 Jul 18;24(14):11574. doi: 10.3390/ijms241411574.

Authors

Deepan Singh¹, Jennifer L Miller², Edward Robert Wassman³, Merlin G Butler⁴, Allison Foley Shenk³, Monica Converse³, Maria Picone³

Affiliations

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 [...].

Publication types

Editorial

MeSH terms

Chromosomes
Chromosomes, Human, Pair 15 / genetics
Drug Approval
Genomic Imprinting
Humans
Prader-Willi Syndrome* / drug therapy
Prader-Willi Syndrome* / genetics