achondroplasia

Jun 03, 2025

Beam’s BEAM-302 Earns FDA Orphan Drug Tag for AATD; Incannex’s IHL-42X Moves to Phase III After FDA Protocol OK; Kura and Kyowa Kirin’s Ziftomenib Gets FDA Priority Review in NPM1-Mutant AML; GSK’s Linerixibat NDA Accepted by FDA for PBC-Linked Pruritus; Ascendis’ TransCon CNP Granted FDA Priority Review for Achondroplasia

Jan 22, 2024

Achondroplasia Treatment Space: Hunt for Potential Curative Therapies

Oct 24, 2023

FDA Approves PENBRAYA for Most Common Serogroups Causing Meningococcal Disease; BIMZELX Approved Moderate to Severe Plaque Psoriasis; FDA Approves BioMarin’s VOXZOGO; FDA Fast Track Designation to ANPD001 for Parkinson’s Disease; UCB Announces FDA Approval of ZILBRYSQ; EMA Granted Orphan Drug Designation to Lisata’s LSTA1

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