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Hot Topics of the Day|PHGKB
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11/03/2019

Hot Topics of the Day are picked by experts to capture the latest information and publications on public health genomics and precision health for various diseases and health topics. Sources include published scientific literature, reviews, blogs and popular press articles.

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Archived Hot Topics of the Day By Date

FDA approves new breakthrough therapy for cystic fibrosis- Treatment approved for approximately 90% of patients with cystic fibrosis, many of whom had no approved therapeutic options
FDA News Release, October 2019

Studies Yield ‘Impressive’ Results in Fight Against Cystic Fibrosis
N Chokshi, NY Times, October 31, 2019

A pair of new studies report “impressive” benefits from a drug therapy for cystic fibrosis, a deadly and devastating disease that affects tens of thousands of people worldwide. These findings indicate that it may soon be possible to offer safe and effective molecularly targeted therapies to 90 percent of persons with cystic fibrosis.

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial
HGM Heijerman et al, Lancet, October 31, 2019

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
PJ Middleton et al, NEJM, October 31, 2019

Polygenic risk scores predict diabetic complications and their response to therapy
J Tremblay et al, MedRXIV, November 1, 2019

This novel polygenic prediction model identified people with diabetes at low and high risk of complications and improved targeting those at greater benefit from intensive therapy while avoiding unnecessary intensification in low-risk subjects.

Predicting Clinical Dementia Rating Using Blood RNA Levels
JB Miller et al, MedRXIV, November 2, 2019

The Clinical Dementia Rating (CDR) is commonly used to assess cognitive decline in Alzheimer's disease patients. We then used machine learning to predict cognitive status using only blood RNA levels. We propose that combining individually suggestive, blood RNA levels can significantly improve diagnostic accuracy.

Year in Review: Ovarian Cancer: PARP inhibitors, genetic testing, and novel combinations
I Ingram, Medpage Today, November 1, 2019

Two themes dominated the ovarian cancer headlines in 2019 -- moving PARP inhibitors into earlier lines of therapy for advanced-stage disease and genetic testing. Over the summer, the U.S. Preventive Services Task Force (USPSTF) broadened its criteria for BRCA cancer susceptibility gene testing in women.


Disclaimer: Articles listed in Hot Topics of the Day are selected by Public Health Genomics Branch to provide current awareness of the scientific literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the Clips, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.
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