Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy

计划入组 50 人 · 首次发布于 2021 年 6 月 23 日

Developmental and Epileptic Encephalopathy of Genetic Etiology: Natural History Through Reuse of Clinical Data

计划入组 400 人 · 首次发布于 2024 年 4 月 23 日

Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy

计划入组 41 人 · 首次发布于 2022 年 11 月 25 日

Developmental and epileptic encephalopathy (Biogen)

STXB1 gene therapy (AAV, developmental and epileptic encephalopathy)

WWOX gene (spinocerebellar ataxia 12/developmental and epileptic encephalopathy)

BROOKDALE UNIVERSITY HOSPITAL MEDICAL CENTER

United States

SaNOtize Research and Development Corp

Canada

Aptevo Research And Development LLC

United States

CAP-002 Receives FDA Orphan Drug Designation for STXBP1-Related Developmental and Epileptic Encephalopathy

Praxis Precision Medicines to Showcase Epilepsy Pipeline at AES 2025 with Full RADIANT Study Results

UCB's Fenfluramine Achieves Positive Phase 3 Results in Ultra-Rare CDKL5 Deficiency Disorder