Ultragenyx

Breaking rare disease news: With the Mikaela Naylon Give Kids a Chance Act signed into law, the Rare Pediatric Disease Priority Review Voucher (PRV) program has been renewed through September 30, 2029.

The PRV program has played an important role in accelerating the development of treatments for children with rare diseases. Its renewal reinforces the path from early research to real options for patients and families, while providing incentives that can help smaller and emerging companies advance promising development programs.

Thank you to the congressional champions including the sponsors, the entire House and Senate leadership for enabling this progress!

We are encouraged by this continued commitment to rare pediatric disease innovation and remain focused on what matters most, delivering meaningful therapies to patients and families who cannot afford to wait.

Today is Creatine Deficiency Day. Like many rare diseases, early diagnosis and intervention can make a real difference.

Throughout the day, the Association for Creatine Deficiencies is highlighting the journeys of families living with Cerebral Creatine Deficiency Syndrome (CCDS), including early signs to look out for. Follow along to hear their stories.