As we close out 2021, please accept our heartfelt thanks to you — the patients, parents, caregivers, and advocates who guide our work at Praxis. We are truly grateful to have you as partners as we travel together on the path of drug discovery and development. We recognize that behind every data point, is a person, an experience, and a voice — thank you for inspiring us with yours. Wishing you happiness and health in the new year.
At Praxis, we’re on a mission to develop novel therapies for SCN2A. We believe collaboration with the SCN2A community is critical in this work. We have compiled some questions we have received from you, along with our responses, to keep you informed on our journey.
We’re pleased to share this special Praxis Precision Medicines SCN2A and SCN8A community update. We invite you to learn more about what Praxis has been working on this Spring / Summer, and we thank you for your insights, interest, and participation.
Praxis has partnered with Ciitizen, an online platform that allows patients and caregivers an opportunity to advance research, to better understand variant characterization within SCN2A-gene mutations through the collection of EEG data from patients around the world. Earlier in 2021, Ciitizen and Praxis began partnering with Ciitizen on an observational study with SCN2A families in the United States and we are excited to expand this offering to families internationally.
At Praxis, we believe that if families and caregivers are a part of the research and development process it will improve our chances to successfully deliver a potentially disease-modifying treatment to patients with mutations in the SCN2A gene.
On Wednesday, April 28, Praxis was granted orphan-drug designation (ODD) by the FDA for PRAX-562 for “treatment of SCN2A developmental and epileptic encephalopathy.”
We would like to extend our sincere gratitude to SCN2A community members who joined us for our virtual community discussions in March. We hope you found the information helpful.
Like you, we at Praxis Precision Medicines (Praxis) envision a future where there is greater understanding of SCN2A-related gene mutations and better treatments to help the children and families affected by this rare disorder.
Exciting news for the SCN2A community! Praxis has recently entered into an innovative research collaboration with The Florey Institute of Neuroscience and Mental Health.
At Praxis, we honor International #SCN2A Awareness Day and remain committed to providing therapies for patients as efficiently as possible.