• Citer

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.

par Voit, Thomas;Topaloglu, Haluk;Straub, Volker;Muntoni, Francesco;Deconinck, Nicolas ;Campion, Giles;De Kimpe, Sjef J;Eagle, Michelle;Guglieri, Michela;Hood, Steve;Liefaard, Lia;Lourbakos, Afrodite;Morgan, Allison;Nakielny, Joanna;Quarcoo, Naashika;Ricotti, Valeria;Rolfe, Katie;Servais, Laurent;Wardell, Claire;Wilson, Rosamund ;Wright, Padraig;Kraus, John E
Référence Lancet neurology, 13, 10, page (987-996)
Publication Publié, 2014-10

Article révisé par les pairs

• Accès en ligne
• Détails
• Contenu
• Statistiques

Titre:	Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.
Auteur:	Voit, Thomas; Topaloglu, Haluk; Straub, Volker; Muntoni, Francesco; Deconinck, Nicolas; Campion, Giles; De Kimpe, Sjef J; Eagle, Michelle; Guglieri, Michela; Hood, Steve; Liefaard, Lia; Lourbakos, Afrodite; Morgan, Allison; Nakielny, Joanna; Quarcoo, Naashika; Ricotti, Valeria; Rolfe, Katie; Servais, Laurent; Wardell, Claire; Wilson, Rosamund; Wright, Padraig; Kraus, John E
Informations sur la publication:	Lancet neurology, 13, 10, page (987-996)
Statut de publication:	Publié, 2014-10
Sujet CREF:	Sciences bio-médicales et agricoles
MeSH keywords:	Adrenal Cortex Hormones -- therapeutic use
	Anti-Inflammatory Agents -- therapeutic use
	Area Under Curve
	Child
	Child, Preschool
	Double-Blind Method
	Dystrophin -- genetics
	Exons
	Female
	Gene Deletion
	Humans
	Male
	Muscular Dystrophy, Duchenne -- drug therapy -- genetics
	Oligonucleotides -- adverse effects -- therapeutic use
	Real-Time Polymerase Chain Reaction
	Treatment Outcome
	Walking
Note générale:	SCOPUS: ar.j
Langue:	Anglais
Identificateurs:	urn:issn:1474-4422
	info:doi/10.1016/S1474-4422(14)70195-4
	info:pii/S1474-4422(14)70195-4
	info:scp/84907991204
	info:pmid/25209738