NATIONAL INSTITUTES OF HEALTH

Symptoms related to sexual health in early-stage breast cancer patients on adjuvant endocrine therapy (AET) were linked to lower adherence in Black women, according to a study presented at the 17th AACR Conference. Both Black and white patients reported lower mental quality of life due to vaginal itchiness, painful intercourse, and loss of interest in sex. Black patients also experienced lower physical quality of life from loss of interest in sex. Vaginal dryness and decreased libido were linked to lower AET adherence in Black women only. The study highlights the need for addressing sexual health symptoms to improve treatment adherence.

The FDA has approved arimoclomol (Miplyffa) for treating Niemann-Pick disease type C (NPC) in patients aged 2 years and older, in combination with miglustat. This approval is based on a phase 2/3 trial showing arimoclomol met its primary outcome measure of improving the NPC Clinical Severity Scale score. Arimoclomol, an orally delivered medication, increases the activation of transcription factors EB and E3, leading to upregulation of lysosomal genes. Zevra Therapeutics plans to launch the therapy in the U.S. within 8 to 12 weeks.

UAMS received a $2.2 million NEI grant for 5 years to study myeloid cells' role in retinopathy, focusing on efferocytosis enhancement for potential therapeutic benefits.

A dermatology team at the University of Miami received a $2.2 million grant to study hidradenitis suppurativa (HS), a painful skin condition. HS lacks an animal model, prompting the team to create the first human model of the disease. The study aims to understand HS's genetic component and develop targeted therapies.

EMA approves Bavarian Nordic's mpox vaccine for adolescents aged 12-17 in Europe, based on NIH-sponsored trial data showing non-inferior immune responses and similar safety profile. Bavarian Nordic plans to expand approval to other high-risk countries, including Africa, where mpox has caused over 25,000 cases and 720 deaths.

FDA approves MIPLYFFA, the first treatment for Niemann-Pick disease type C (NPC), in combination with miglustat for neurological manifestations in adults and pediatric patients 2 years and older. Zevra Therapeutics receives a rare pediatric disease priority review voucher and launches AmplifyAssist™ patient support program. Conference call scheduled for Sept. 23, 2024.

RIPPLES SEP plans a 2-week summer program for AI/AN high school students to inspire future scientists, change makers, and community health leaders, focusing on culturally relevant research and mentoring.