Track FDA orphan designations, clinical trials, patents, and competitor moves across rare disease drug development — all in one monthly intelligence report with real-time alerts.
FDA granted 14 new orphan drug designations this month, including 3 in neuromuscular diseases.
+14 newPhase 2 readout for CLZ-2002 (CMT) expected Q3. New Phase 1 initiated for HGPS combination therapy.
8 updatesNew use-patent filed for Sirolimus + mTOR modulator combination in progeroid syndromes.
IP alertHealx announces $47M Series C. Recursion partners with Bayer for rare genetic diseases.
5 movesNature Medicine publishes gene therapy results for DMD. 12 new repurposing studies indexed.
12 papersORPHAN Cures Act implementation update. New FDA guidance on rare disease clinical endpoints.
Policy shift30+ page comprehensive brief covering all rare disease drug development activity from the past month.
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Complete database of active orphan designations, approvals, and pending applications — updated weekly.
Rare disease trials tracked across all phases. New initiations, completions, terminations, and readouts.
New filings, grants, and expirations for rare disease use-patents. Freedom-to-operate signals.
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