UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults.

Introduction: 2022 was a landmark year with two adeno-associated viral vectors (AAVs) receiving conditional marketing authorization from EMA for the treatment of persons with severe haemophilia A and severe to moderately severe haemophilia B and a third in 2024. Gene therapy is a transformative, irreversible treatment with long-lasting effects, necessitating development of new clinical pathways to ensure optimal outcomes.

Aim: To develop a consensus framework and service specification for delivery of AAV gene therapy for haemophilia in adults within the UK using the hub-and-spoke model proposed by the European Association of Haemophilia and Allied Disorders and the European Haemophilia Consortium.

Mediating effects of inequitable gender norms on intimate partner violence and contraceptive use in a cluster randomized control trial in Niger.

Previous research has demonstrated that the Reaching Married Adolescents intervention (RMA) was associated with changes in inequitable gender norms, intimate partner violence (IPV), and modern contraceptive use. This study seeks to understand if changes in inequitable gender norms mediate the RMA intervention's effects on contraceptive use and intimate partner violence (IPV). A four-arm cluster randomized control trial was conducted to evaluate effects of the RMA intervention (household visits, small groups, combination, control) on married adolescent girls and their husbands in Dosso, Niger (baseline: 1042 dyads; 24m follow-up: 737 dyads; 2016-2019).

Speaker-independent speech inversion for recovery of velopharyngeal port constriction degreea).

For most of his illustrious career, Ken Stevens focused on examining and documenting the rich detail about vocal tract changes available to listeners underlying the acoustic signal of speech. Current approaches to speech inversion take advantage of this rich detail to recover information about articulatory movement. Our previous speech inversion work focused on movements of the tongue and lips, for which "ground truth" is readily available.

Computer-assisted syllable analysis of continuous speech as a measure of child speech disordera).

In this study, a computer-driven, phoneme-agnostic method was explored for assessing speech disorders (SDs) in children, bypassing traditional labor-intensive phonetic transcription. Using the SpeechMark® automatic syllabic cluster (SC) analysis, which detects sequences of acoustic features that characterize well-formed syllables, 1952 American English utterances of 60 preschoolers were analyzed [16 with speech disorder present (SD-P) and 44 with speech disorder not present (SD-NP)] from two dialectal areas. A four-factor regression analysis evaluated the robustness of seven automated measures produced by SpeechMark® and their interactions.

Physical, Sexual, and Intimate Partner Violence Among Transgender and Gender-Diverse Individuals.

Importance: Transgender and gender-diverse (TGD) communities experience disproportionate levels of violence, yet due to limitations in measuring TGD identity, few state-representative estimates are available.

Objective: To assess gender identity differences in experiences of violence among adults.

Design, Setting, And Participants: Cross-sectional data from the 2023 California Violence Experiences (CalVEX) survey, weighted to provide state-representative estimates, was used to assess associations between gender identity and past-year experiences of violence among adults 18 years and older.