NATIONAL INSTITUTES OF HEALTH

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.

Researchers at Johns Hopkins Kimmel Cancer Center found MYC gene activation initiates prostate cancer, initially attracting immune cells but later evading them, suggesting MYC as a therapeutic target.

FDA approves Miplyffa (arimoclomol) for Niemann-Pick disease type C (NPC), the first NPC drug. Indicated for neurological manifestations in adult and pediatric patients 2 years and older, in combination with miglustat. Zevra Therapeutics receives rare pediatric disease priority review voucher.