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Givosiran: A Targeted Treatment for Acute Intermittent Porphyria

Program: Education Program
Session: Integrating New Therapies into the Management of Classical Heme Disorders
Hematology Disease Topics & Pathways:
Research, Clinical trials, Adult, Translational Research, Drug development, Clinical Research, Genetic Disorders, Diseases, Real-world evidence, Treatment Considerations, Adverse Events, Metabolic Disorders, Study Population, Human
Saturday, December 7, 2024, 2:00 PM-3:15 PM

Amy Dickey, MD

Massachusetts General Hospital, Boston, MA

Disclosures: Dickey: Disc Medicine: Other: Principal Investigator on clinical trial.

OffLabel Disclosure: Much of porphyria treatment is off-label, so I plan to mention some off label uses of both hemin and givosiran

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