Myrtelle Announces Positive 6-month Post-Treatment Data in Patients in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease
Analyses at 6 months showed increases in myelin, white matter and other MRI and MRS measurements as well as functional improvements on validated assessment scales Encouraging initial efficacy data…
Article Date
April 20, 2023
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January 26, 2026
Understanding the US FDA’s Special Designations and Approval Pathways
Recognizing the challenges of drug development, the US FDA has established several special designations and…
January 26, 2026
Understanding the US FDA’s Special Designations and Approval Pathways
Recognizing the challenges of drug development, the US FDA has established several special designations and…