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CRYSVITA Mechanism of Action 

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DICERNA: Mechanism of action of GalXC RNAi therapy

medical illustration of This mechanism of action illustration explains how the CRYSVITA molecule binds to FGF23 to normalize phosphorous levels in the bloodstream. CRYSVITA (burosumab-twza) is the only FDA-approved therapy targeting the underlying cause of XLH in adults and children 6 months or older. Client: Ultragenyx Pharmaceuticals
Next medical illustration of This is a MOD graphic of ADA-SCID (Adenosine deaminase severe combined immune deficiency), an ultra-rare inherited genetic disorder. It is caused by a deficiency in the adenosine deaminase (ADA) enzyme and is often fatal if untreated. ADA deficiency increases the risk of severe and recurring infections by inducing apoptosis in B, T, and NK lymphocytes through toxic buildups of deoxyadenosine. Client: Leadiant Biosciences

ADA-SCID Mechanism of Disease

This mechanism of action illustration explains how the CRYSVITA molecule binds to FGF23 to normalize phosphorous levels in the bloodstream. CRYSVITA (burosumab-twza) is the only FDA-approved therapy targeting the underlying cause of XLH in adults and children 6 months or older. Client: Ultragenyx Pharmaceuticals

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