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http://dx.doi.org/10.1056/NEJMe2030687 | DOI Listing |
J Neurodev Disord
January 2025
Rett Syndrome Research Trust, Trumbull, CT, USA.
Background: Preclinical studies and anecdotal case reports support the potential therapeutic benefit of low-dose oral ketamine as a treatment of clinical symptoms in Rett syndrome (RTT); however, no controlled studies have been conducted in RTT to evaluate safety, tolerability and efficacy.
Design: This was a sequentially initiated, dose-escalating cohort, placebo-controlled, double blind, randomized sequence, cross-over study of oral ketamine in 6-12-year-old girls with RTT to evaluate short-term safety and tolerability and explore efficacy.
Methods: Participants were randomized to either five days treatment with oral ketamine or matched placebo, followed by a nine-day wash-out period and then crossed-over to the opposite treatment.
Endocr Pract
January 2025
University of Colorado Anschutz Medical Campus, Barbara Davis Center for Diabetes, 1775 Aurora Court, Aurora, CO 80045, USA. Electronic address:
Objectives: Type 1 diabetes (T1D) in pregnancy is challenging. This study explores how assisted hybrid closed-loop (HCL) therapy versus sensor-augmented pump therapy (SAPT) impacts quality of life (QoL) in pregnancy.
Methods: We interviewed 22 of 24 participants randomized to HCL therapy or SAPT in the Pregnancy Intervention with a Closed-Loop System study.
ALTEX
January 2025
F. Hoffmann-La Roche Ltd, Precision Safety, Product Development, Basel, Switzerland.
The regular workshops held by the Center for Alternatives to Animal Testing (CAAT) on biology-inspired microphysiological systems (MPS) taking place every four years, have become a reliable measure to assess fundamental scientific, industrial and regulatory trends for translational science in the MPS-field from a bird's eye view. The 2023 workshop participants at that time concluded that the technology as used within academia has matured significantly, underlined by the broad use of MPS and the steadily increasing number of high quality research publications - yet, broad industry adoption of MPS has been slow, despite strong interest. Academic research using MPS primarily aims to accurately recapitulate human biology in MPS-based organ models in areas where traditional models have been lacking key elements of human physiology, thereby enabling breakthrough discoveries for life sciences.
View Article and Find Full Text PDFJAMA
January 2025
Assistant Secretary for Technology Policy/Office of the National Coordinator for Health IT, Washington, DC.
Importance: Health information technology, such as electronic health records (EHRs), has been widely adopted, yet accessing and exchanging data in the fragmented US health care system remains challenging. To unlock the potential of EHR data to improve patient health, public health, and health care, it is essential to streamline the exchange of health data. As leaders across the US Department of Health and Human Services (DHHS), we describe how DHHS has implemented fundamental building blocks to achieve this vision.
View Article and Find Full Text PDFPhysiol Rev
January 2025
Department of Investigative Medicine, Imperial College London, Hammersmith Hospital, London, United Kingdom.
Kisspeptin and neurokinin B (NKB) play a key role in several physiological processes including in puberty, adult reproductive function including the menstrual cycle, as well as mediating the symptoms of menopause. Infundibular kisspeptin neurons, which co-express NKB, regulate the activity of gonadotropin releasing hormone (GnRH) neurons, and thus the physiological pulsatile secretion of GnRH from the hypothalamus. Outside of their hypothalamic reproductive roles, these peptides are implicated in several physiological functions including sexual behavior and attraction, placental function, and bone health.
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