The CDC is seeking public comments on the Traveler-Based Genomic Surveillance Program's Traveler Questionnaire, aimed at enhancing data collection efforts. This initiative is part of the agency's commitment to reduce public burden while maximizing the utility of government information.

The CDC is seeking public comment on a proposed data collection project titled Land Activity Illness and Death Reporting. This initiative aims to enhance the utility of government information while reducing public burden, as mandated by the Paperwork Reduction Act.

FDA has approved Zycubo (copper histidinate) as the first treatment for Menkes disease in pediatric patients. This landmark approval addresses a critical need for a condition that affects copper metabolism in children.

The rare disease community is intensifying efforts to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, aiming to pass the Give Kids a Chance Act by 2026. This legislation is crucial for ensuring access to lifesaving treatments for children with rare diseases.

The FDA has released draft guidance to modernize statistical methods in clinical trials, emphasizing the use of Bayesian methodologies. This initiative aims to enhance data utilization and improve trial efficiency, ultimately benefiting drug developers and patient outcomes.

The RealiseD project, introduced by Patient Engagement Manager Rita Francisco, aims to enhance clinical trial innovation for rare diseases. This initiative seeks to improve patient engagement and streamline processes in clinical research.

The FDA has released a draft guidance on the use of Bayesian methodology in clinical trials for drug and biological products. This guidance aims to clarify the application of Bayesian methods to enhance the effectiveness and safety assessments in pivotal trials.

The FDA has announced a more flexible approach to overseeing CMC requirements for cell and gene therapies, aiming to foster innovation in this rapidly evolving field. This change is expected to streamline the development process for CGTs, enhancing access to new treatments.

The EveryLife Foundation for Rare Diseases has appointed a new Chair and two new members to its Board of Directors, enhancing its leadership as it continues to advocate for rare disease policies. This transition marks a strategic move to strengthen the foundation's impact in the rare disease community.

Aktis Oncology successfully raised $318 million in its IPO, exceeding expectations and marking the largest biotech IPO of 2026 to date. The company plans to allocate $140 million to $150 million of the proceeds for ongoing U.S. development of its drug Ac-AKY-1189.

Aktis has successfully raised $318 million in its IPO, marking one of the largest biotech stock offerings in two years, aided by partner Eli Lilly. This follows $346 million in private funding aimed at advancing its pipeline of radiopharmaceuticals.

The Administration for Children and Families proposes a new data collection for evaluating the Trafficking Victim Assistance Program and Aspire. This evaluation aims to assess program characteristics, implementation challenges, and successes.

Elisabeth Gardiner, CSO of Tevard Biosciences, highlights the potential of transfer RNA in advancing gene therapies for rare diseases. She notes that traditional gene therapy methods face challenges in addressing large structural proteins, emphasizing the need for innovative approaches in this field.

Antje Enekwe shares her experiences and challenges of raising a child with Smith-Lemli-Opitz syndrome, highlighting the resilience required in such circumstances. This personal narrative contributes to awareness and understanding of the daily realities faced by families affected by this rare disease.

Emerging healthcare companies are gearing up for IPOs in 2025, with a focus on digital health innovations. Notably, Aptar Digital Health is partnering with AstraZeneca to develop AI algorithms for chronic kidney disease detection, while Wellgistics Health aims to enhance patient outcomes through integrated digital health solutions.