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rare pediatric disease

2 articles
BenzingaBenzinga··Atossa Therapeutics Inc

Atossa Therapeutics Advances (Z)-Endoxifen Pipeline With FDA Orphan Designations

Atossa Therapeutics reported Q1 2026 expenses of $9.9M, secured FDA orphan drug designation for lead candidate (Z)-endoxifen in rare diseases.
ATOSclinical developmentbreast cancer
GlobeNewswire Inc.GlobeNewswire Inc.··Ascendis Pharma A/S

Ascendis Pharma Wins FDA Clearance for First Weekly Achondroplasia Treatment in Children

Ascendis Pharma wins FDA approval for YUVIWEL, the first once-weekly treatment for pediatric achondroplasia. The therapy offers improved dosing convenience over existing options.
ASNDFDA approvalaccelerated approval