BOSTON, Nov. 16, 2023 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for ...

The SCN2A Foundation today announced a research collaboration with Unravel Biosciences, Inc., an AI-enabled therapeutics company established to advance drugs for complex diseases. The collaboration ...

Clear recognition of high unmet need and urgency for the SCN2A community and plausibility of the mechanism of elsunersen FDA agreed to proposed changes to the EMBRAVE3 trial design to be a single-arm, ...

In the study, the UCSF team demonstrated that restoring healthy levels of SCN2A expression through CRISPR activation, even in late development, could reverse several disease-relevant deficits present ...

A genetic change or variant in a gene called SCN2A is a known cause of infantile seizures, autism spectrum disorder and intellectual disability, as well as a wide range of other moderate-to-profound ...

Two new drug applications (NDA) for ulixacaltamide in essential tremor (ET) and for relutrigine in SCN2A and SCN8A ...

A technology that activates healthy genes reduced seizures in a mouse model of a severe genetic neurological condition (1 Trusted Source Can CRISPR Fix a Childhood Brain Disorder? Go to source). When ...

The Breakthrough Therapy Designation (BTD) was granted based on the highly compelling results from the Phase 2 EMBOLD trial in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) The ...

Topline study results to be shared at the American Epilepsy Society Annual Meeting on December 6, 2025 Praxis confirms an upcoming meeting with the FDA to discuss the results and NDA path BOSTON, Dec.

CAMBRIDGE, Mass., April 30, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of ...

Could CRISPR Fix this Severe Childhood Brain Disorder? A technology that activates healthy genes reduced seizures in a mouse model of a severe genetic neurological condition. When brain development ...