Full Press Release Details
Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM).
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Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
Key Takeaway: Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
BiopharmaWatch Analysis
From our catalyst data and publicly available data · not financial adviceBest trade, last catalyst
+43%
120-day peak, hindsight
Typical move
2.9%
average across 6 past catalysts
Cash runway
~13 mo
Low dilution risk
Lead asset
BPS804
Phase 2 · Hypophosphatasia