The U.S. Food and Drug Administration today announced six additional awardees under the Commissioner’s National Priority Voucher (CNPV) pilot program.

The U.S. Food and Drug Administration issued 18 warning letters to owners of websites illegally marketing unapproved and misbranded botulinum toxin products, commonly called Botox.

11 people from 7 states have gotten sick from the same strain of Salmonella.

CDC urges people to check their refrigerators and freezers for recalled foods. Do not eat them.

EURORDIS celebrates the 40th workshop of its Round Table of Companies, focusing on the integration of patient experience data into medicines regulation. This initiative aims to enhance the role of patient insights in the drug development process.

Global Genes hosted the RARE Advocacy Exchange Session 8 on October 16, 2025, focusing on educating patients about their rights in the rare disease landscape. This session aims to empower individuals and organizations advocating for rare disease awareness and support.

The EveryLife Foundation has submitted comments to the FDA on the Reauthorization of Medical Device User Fee Agreements (MDUFA) for fiscal years 2028 and beyond. This initiative could impact the regulatory landscape for medical devices affecting rare diseases.

Over 190 organizations, led by the EveryLife Foundation, support the Give Kids a Chance Act of 2025, which aims to reauthorize the Rare Pediatric Disease Priority Review Voucher program for five years. This legislation is crucial for accelerating the development of treatments for rare pediatric diseases.

The EU Biotech Act is poised to significantly impact rare disease progress, with EURORDIS outlining key priorities for its implementation. Stakeholders are urged to ensure that the Act translates into tangible benefits for the rare disease community.

CDC updated updated its adult and child immunization schedules to apply individual-based decisions

Global Genes releases a Quick Guide aimed at helping advocates establish nonprofit patient advocacy organizations. This resource addresses key challenges and provides actionable insights for those looking to make a difference in the rare disease community.

The U.S. government entered a shutdown on October 1 due to Congress failing to pass appropriations for the 2026 fiscal year. The duration of the shutdown remains uncertain, potentially impacting federal operations and funding for various programs.

The RARE Advocacy Exchange Session 7 focused on the importance of obtaining precise genetic diagnoses for rare diseases. This session, held on September 18, 2025, highlights ongoing efforts to improve patient outcomes through better genetic understanding.

We extend our deepest condolences to Suzy's family, her friends, and all who cared about her.

CDC Expands Listeria Outbreak Alert to Include Additional Prepared Foods

The CDC has expanded its investigation into a Listeria outbreak, now reporting 3 additional illnesses and 1 more death linked to prepared meals. This raises awareness about food safety and the importance of monitoring outbreaks.

New CDC data shows U.S. cases of newborn syphilis increased for the 12th consecutive year in 2024

Global Genes hosts the RARE Advocacy Exchange Session 6, focusing on the unique grief associated with rare disease diagnoses. This session aims to provide support and understanding for those affected by the emotional challenges of living with rare conditions.

A CDC report found a dramatic increase in a dangerous type of drug-resistant bacteria.

A student shares insights from the 2025 RARE Drug Development Symposium, highlighting key lessons on collaboration and innovation in rare disease drug development. The perspective emphasizes the importance of engaging young minds in the conversation around rare diseases.