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Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. The most common form of CAH, 21-hydroxylase deficiency, disrupts hormone production, leading to cortisol deficiency and an overproduction of androgens.

Genetics 116
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Exercise hormone may modulate genes associated with replication of novel coronavirus

Scienmag

Brazilian researchers observed that in uninfected adipocytes, the hormone irisin altered the expression of genes that regulate ACE-2, which encodes a protein to which the virus binds in order to invade human cells.

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Genetically modified mice pave the way for customized medicine in a rare disease

Medical Xpress

Ana Guadaño at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior of the cell.

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Adyn Test Brings Precision Medicine to Birth Control Prescription Choices

BioSpace

This is an effort to eliminate severe adverse reactions by matching the birth control to the genetics and hormones of the individual.

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Can the FDA keep the momentum going for rare disease drug approvals?

Pharmaceutical Technology

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. The mutation leads to disordered thyroid hormone transport, which causes patients to experience intellectual and motor disabilities. T3 is a major hormone in the blood that regulates thyroid levels.

Drugs 317
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Patient Safety and Radiopharmaceuticals Administration: Minimizing Radiation Exposure and Risks

Worldwide Clinical Trials

In nuclear medicine, radiopharmaceuticals play a pivotal role in two primary applications: Diagnostic Imaging Radiopharmaceuticals are administered to patients, where they accumulate in specific organs or tissues of interest.

Hormones 130
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Veozah (Fezolinetant) Is a New Type of Drug for Menopausal Hot Flashes

XTalks

. “Today’s approval of fezolinetant is a significant and long-awaited milestone for individuals in the US who experience moderate to severe vasomotor symptoms during the menopausal transition,” said Genevieve Neal-Perry, MD, PhD, Chair of the UNC School of Medicine Department of Obstetrics and Gynecology, in Astellas’ news release.