A randomized, placebo-controlled, cross-over trial of ketamine in Rett syndrome.

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.

MYC and HSF1 Cooperate to Drive Sensitivity to Polo-like Kinase 1 Inhibitor Volasertib in High Grade Serous Ovarian Cancer.

Ovarian cancer is a deadly gynecological disease with frequent recurrence. Current treatments for patients include platinum-based therapy regimens with PARP inhibitors specific for HR-deficient high-grade serous ovarian cancers (HGSOCs). Despite initial effectiveness, patients inevitably develop disease progression as tumor cells acquire resistance.

What About Nights, Weekends, and Wait Times? Adding an On-Demand Facilitated Telemedicine Buprenorphine Component to a Community-Based Peer Support Outreach Program.

Background: In 2020, loosened federal regulations allowed for buprenorphine for opioid use disorder to be initiated via telemedicine. In response to these regulatory changes and growing racial inequities in overdose in St. Louis, MO, a local, peer-led outreach program incorporated a new rapid access (RA) to buprenorphine program.

The urgent need to accelerate synthetic data privacy frameworks for medical research.

Synthetic data, generated through artificial intelligence technologies such as generative adversarial networks and latent diffusion models, maintain aggregate patterns and relationships present in the real data the technologies were trained on without exposing individual identities, thereby mitigating re-identification risks. This approach has been gaining traction in biomedical research because of its ability to preserve privacy and enable dataset sharing between organisations. Although the use of synthetic data has become widespread in other domains, such as finance and high-energy physics, use in medical research raises novel issues.

Parent-Reported Outcome Measures for Individuals with Fragile X Syndrome: Clinically Meaningful Change Thresholds.

Estimating meaningful change thresholds (MCT) on clinical outcome assessments is an important consideration when evaluating treatments. In fragile X syndrome (FXS) research, there has been no consensus on how to define MCT's on several commonly used outcome measures. The purpose of the current study was to determine clinically relevant MCT's of caregiver-rated assessments using data from a phase 3 clinical trials of arbaclofen (Berry-Kravis et al.