Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trials

Existing clinical tools that measure non-seizure outcomes lack the range and granularity needed to capture skills in developmental and epileptic encephalopathy (DEE)-affected individuals who also fall in the severe to profound range of intellectual disability. This effectively excludes those with se...

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Veröffentlicht in:	Therapeutic advances in rare disease 2024-01, Vol.18, p.26330040241249762
Hauptverfasser:	Hecker, JayEtta, Conecker, Gabrielle, Chapman, Chere, Hommer, Rebecca, Ludwig, Natasha N, Sevinc, Gunes, Te, Sara, Wojnaroski, Mary, Downs, Jenny, Berg, Anne T
Format:	Artikel
Sprache:	eng
Schlagworte:	Roadmaps and Resources to Advance Therapy Development for Rare Epilepsies
Online-Zugang:	Volltext
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