perspective on unmet need for effective treatment in fibrous dysplasia and rare paediatric bone disease

Original title: “Perspective on unmet need for effective treatment in fibrous dysplasia and rare paediatric bone disease.”

consensus recommendations for next generation sequencing based genetic testing in rare neurological diseases

Original title: “Consensus recommendations for next-generation sequencing-based genetic testing in Rare Neurological diseases.”

The Food and Drug Administration (FDA) is amending a notice entitled "Fee Rate for Using a Priority Review Voucher in Fiscal Year 2026" that appeared in the Federal Register on September 18, 2025. The notice established the FY 2026 priority review fee rate applicable to submission of eligible applications for review of human drug or biological products using a rare pediatric disease, material threat medical countermeasure, or tropical disease priority review voucher and outlined the payment proc

Original title: “Fee Rate for Using a Priority Review Voucher in Fiscal Year 2026; Correction”

reopening the silk road for rare neurological diseases

Original title: “Reopening the Silk Road for rare neurological diseases.”