BioMarin Files Vosoritide For Achondroplasia, But Investors Still Upset About Gene Therapy Setback
Vosoritide, a once daily injection, could be the first drug available for achondroplasia, the most common form of dwarfism.
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R&D president Hank Fuchs said vosoritide can be filed based on height gains for children and safety in babies and toddlers with the most common form of dwarfism, but timing depends on meetings with regulators.