CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.K.
Denali Therapeutics Inc. announced positive long-term data from its Phase 1/2 study of tividenofusp alfa (DNL310) in patients with Hunter syndrome (MPS II), showing sustained reductions in key disease ...
Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier for neurodegenerative diseases and lysosomal storage ...
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California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’
A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...
The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for tividenofusp alfa for the treatment of Hunter syndrome, also known as ...
A Prescription Drug User Fee Act target date of November 9, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...
Mucopolysaccharidosis II is currently treated with an enzyme replacement therapy that does not cross the blood-brain barrier and thus does not address the condition's neurologic consequences. This ...
Additional biomarker data up to two years of treatment continue to demonstrate rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function ...
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