News Related to Glanzmann Thrombasthenia

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January and March 2026 drug pipeline updates span Phase 3, Fast Track and Priority Review

Apr 23, 2026

January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.

Rare Disease Day Highlights Diagnostic Delays and Access Barriers to Gene Therapy

Feb 28, 2026

International Rare Disease Day on February 28 underscores challenges in diagnosing and treating rare genetic conditions, with patients facing years-long diagnostic delays and geographic barriers to accessing gene therapies.