Adrabetadex

Generic Name: Adrabetadex

Brand Names: -

Drug Type: Small Molecule

Chemical Formula: C_₅₄H_₉₄O_₃₈

CAS Number: 128446-35-5

Unique Ingredient Identifier: 8W6Q67R6NX

Background: Adrabetadex is under investigation in clinical trial NCT03887533 (Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1).

Associated Conditions: -

Associated Therapies: -

Clinical Trials

'Light at the end of the tunnel': Family relieved after FDA approves two treatments for rare disease

9-year-old Abby Alvey, diagnosed with Niemann-Pick Disease Type C, has shown resilience and strength. Her participation in a clinical trial for Adrabetadex halted symptom progression. The FDA's approval of two drugs for NPC treatment marks a milestone, offering hope for Abby and others. The Alvey family remains committed to finding a cure, staying #AbbyStrong.

Drug Development Updates

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