Last update 24 May 2025

BCKDH Deficiency (Clinic for Special Children)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms-
Target
BCKDHA x BCKDHB
Action
modulators
Mechanism
BCKDHA modulators(branched chain keto acid dehydrogenase E1 subunit alpha modulators), BCKDHB modulators(branched chain keto acid dehydrogenase E1 subunit beta modulators)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Maple Syrup Urine Disease
Inactive Indication-
Originator Organization
The Clinic for Special Children, Inc.The Clinic for Special Children, Inc.
Active Organization
The Clinic for Special Children, Inc.The Clinic for Special Children, Inc.
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with BCKDH Deficiency (Clinic for Special Children)
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100 Translational Medicine associated with BCKDH Deficiency (Clinic for Special Children)
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100 Patents (Medical) associated with BCKDH Deficiency (Clinic for Special Children)
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100 Deals associated with BCKDH Deficiency (Clinic for Special Children)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Maple Syrup Urine DiseasePreclinical
United States
The Clinic for Special Children, Inc.The Clinic for Special Children, Inc.
15 May 2025
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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Deal

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Clinical Trial

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Approval

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Regulation

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