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6,837 results for “ABCC6 Deficiency”

Trials

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Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency

38 participants · First posted May 4, 2020

Natural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 Deficiency

23 participants · First posted Aug 4, 2026

Active, not recruiting

PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency

54 participants · First posted Mar 8, 2024

Drugs

View all 141 →

CDKL5 deficiency disorder therapeutic program (Vyant Bio Inc/Cyclica Inc)

WWOX gene therapy(Mahzi Therapeutics/ Hebrew University)

CBS-deficiency(Mitorx Therapeutics)

Organizations

Rockwell Medical

United States

Ai-Media Technologies Ltd

United States

News

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Inozyme's INZ-701 Shows Promise in ENPP1 and ABCC6 Deficiencies

BioMarin Awaits Pivotal INZ-701 Trial Results Following $270M Inozyme Acquisition

BioMarin Acquires Inozyme Pharma for $270 Million, Adding Late-Stage Enzyme Therapy for Rare Genetic Disorder

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