Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the doubl...
Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial
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Author / Creator
Marigold Trial Group , Knight, Elia M Pestana , Amin, Sam , Bahi-Buisson, Nadia , Benke, Tim A , Cross, J Helen , Demarest, Scott T , Olson, Heather E , Specchio, Nicola , Fleming, Thomas R , Aimetti, Alex A , Gasior, Maciej , Devinsky, Orrin , Belousova, Elena , Belyaev, Oleg , Ben-Zeev, Bruria , Brunklaus, Andreas , Ciliberto, Michael A. , Darra, Francesca , Davis, Ronald , De Giorgis, Valentina , Doronina, Olga , Fahey, Michael , Guerrini, Renzo , Heydemann, Peter , Khaletskaya, Olga , Lisewski, Pawel , Marsh, Eric D. , Moosa, Ahsan N. , Perry, Scott , Philip, Sunny , Rajaraman, Rajsekar R. , Renfroe, Ben , Saneto, Russell P. , Scheffer, Ingrid E. , Sogawa, Yoshimi , Suter, Bernhardt , Sweney, Matthew T. , Tarquinio, Daniel , Veggiotti, Pierangelo , Wallace, Geoff , Weisenberg, Judy , Wilfong, Angus , Wirrell, Elaine C. , Zafar, Muhammad and Zolnowska, Marta
Publisher
England: Elsevier Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: Elsevier Ltd
Subjects
More information
Scope and Contents
Contents
CDKL5 deficiency disorder (CDD) is a rare, X-linked, developmental and epileptic encephalopathy characterised by severe global developmental impairment and seizures that can begin in the first few months after birth and are often treatment refractory. Ganaxolone, an investigational neuroactive steroid, reduced seizure frequency in an open-label, ph...
Alternative Titles
Full title
Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial
Authors, Artists and Contributors
Author / Creator
Knight, Elia M Pestana
Amin, Sam
Bahi-Buisson, Nadia
Benke, Tim A
Cross, J Helen
Demarest, Scott T
Olson, Heather E
Specchio, Nicola
Fleming, Thomas R
Aimetti, Alex A
Gasior, Maciej
Devinsky, Orrin
Belousova, Elena
Belyaev, Oleg
Ben-Zeev, Bruria
Brunklaus, Andreas
Ciliberto, Michael A.
Darra, Francesca
Davis, Ronald
De Giorgis, Valentina
Doronina, Olga
Fahey, Michael
Guerrini, Renzo
Heydemann, Peter
Khaletskaya, Olga
Lisewski, Pawel
Marsh, Eric D.
Moosa, Ahsan N.
Perry, Scott
Philip, Sunny
Rajaraman, Rajsekar R.
Renfroe, Ben
Saneto, Russell P.
Scheffer, Ingrid E.
Sogawa, Yoshimi
Suter, Bernhardt
Sweney, Matthew T.
Tarquinio, Daniel
Veggiotti, Pierangelo
Wallace, Geoff
Weisenberg, Judy
Wilfong, Angus
Wirrell, Elaine C.
Zafar, Muhammad
Zolnowska, Marta
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2651692888
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2651692888
Other Identifiers
ISSN
1474-4422,1474-4465
E-ISSN
1474-4465
DOI
10.1016/S1474-4422(22)00077-1
