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Patients & Community
Zogenix is working to develop therapies with the potential to transform the lives of patients and their families living with serious rare diseases.
Dravet Syndrome
A rare and severe epilepsy
Dravet syndrome is a rare, severe, treatment-resistant epilepsy syndrome with onset in infancy and serious neurodevelopmental, motor, cognitive, and behavioral consequences that persist into adulthood. Studies have reported an incidence rate for Dravet syndrome of approximately one per 16,000 live births with onset occurring within the first year of life.
- Clinical Studies – Zogenix is not currently enrolling patients in studies for Dravet syndrome.
- EAP – We offer a limited expanded access program (also known as “compassionate use” or “early access”) for our investigational therapy ZX008. Requests for access to experimental therapies must be made by a qualified and licensed physician. Learn More
- Disease – For more information about the US Dravet Syndrome Foundation, click here. For more information about the Dravet Syndrome European Federation, click here.
- Additional Resources – For more information about Dravet Syndrome, please see the Dravet Syndrome, and Dravet Syndrome Caregiver factsheets
Lennox-Gastaut Syndrome (LGS)
A rare and severe epilepsy
LGS is also a form of rare, severe form of epilepsy marked by frequent and prolonged seizures, with peak onset between ages 3 and 5. LGS impacts approximately 14,500 to 18,500 children in the U.S.
- Clinical Studies – Zogenix is not currently enrolling patients in studies for LGS.
- Disease – For more information about the US LGS Foundation, please visit www.lgsfoundation.org.
- Additional Resources – For more information about LGS, please see the LGS factsheet.
CDKL5 Deficiency Disorder (CDD)
A rare and severe epilepsy
CDKL5 deficiency disorder is a rare developmental epileptic encephalopathy caused by mutations in the CDKL5 gene which can manifest in a broad range of clinical symptoms and severity. The hallmarks are early-onset, intractable epilepsy and neurodevelopmental delay impacting cognitive, motor, speech, and visual function. Although rare, the occurrence is believed to be ~1:40,000 -60,000 live births, making it one of the most common forms of genetic epilepsy.[i]
- Clinical Studies – Zogenix is currently enrolling patients for a clinical studies CDD. For more information: https://www.CDDstudy.com
- Registries & Natural History Studies – To help advance CDKL5 research and gain better insights on your child or loved one with CDKL5 Deficiency Disorder, please consider signing up to help the researchers: https://www.cdkl5.com/help-our-researchers/.
- Disease – For more information about CDKL5 deficiency disorder, please visit the International Foundation for CDKL5 Research (IFCR) or the LouLou Foundation.
TK2 Deficiency
A progressive, devastating mitochondrial disease.
Thymidine kinase 2 deficiency (TK2d) is a genetic disorder that results in mitochondrial dysfunction, leading to inadequate energy production in cells. TK2d onset occurs across all ages and causes progressive and severe muscle weakness, respiratory insufficiency, loss of motor function, and is often fatal. There are currently no approved therapies for this disease.
- Clinical Studies – Zogenix is not yet enrolling for Phase 3 trials of MT1621, its investigational treatment for TK2 deficiency.
- Registry – Take on TK2D –Whether you’re living with an MDS, have been diagnosed with TK2d, or are a parent, caregiver, or healthcare professional (HCP) who wants to learn more about MDS and TK2d, join the registry so we can all take on TK2d together.
- Disease – For more information about TK2 deficiency, please visit tk2d.com. Or visit the United Mitochondrial Disease Foundation at https://www.umdf.org/
- Additional Resources – Understanding TK2d