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

Deepan SinghJennifer L MillerEdward Robert Wassman Merlin G ButlerAllison Foley ShenkMonica Converse Maria Picone

Published in: International journal of molecular sciences (2023)

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

Keyphrases

growth hormone
genome wide
case report
copy number
emergency department
mass spectrometry
adverse drug
liquid chromatography