Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial

Zielen, Stefan; Crawford, Thomas; Benatti, Luca; Magnani, Mauro; Kieslich, Matthias; Ryan, Monique; Meyts, Isabelle; Gulati, Sheffali; Borgohain, Rupam; Yadav, Ravi; Pal, Pramod; Hegde, Anaita; Kumar, Suresh; Venkateswar, Anand; Udani, Vrajesh; Vinayan, Kollencheri P; Nissenkorn, Andreea; Fazzi, Elisa; Leuzzi, Vincenzo; Stray-Pedersen, Asbjørg; Pietrucha, Barbara; Pascual, Samuel I; Gouider, Riadh; Koenig, Mary Kay; Wu, Steve; Perlman, Susan; Thye, Dirk; Janhofer, Guenter; Horn, Biljana; Whitehouse, William; Lederman, Howard

doi:10.1016/s1474-4422(24)00220-5

The Lancet Neurology

2024

DOI: 10.1016/s1474-4422(24)00220-5

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Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial

Stefan Zielen,

Thomas Crawford,

Luca Benatti

et al.

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Lessons from the ATTeST trial in ataxia telangiectasia

Koike,

Onodera

2024

The Lancet Neurology

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Lessons from the ATTeST trial in ataxia telangiectasia

Koike,

Onodera

2024

The Lancet Neurology

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Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial

Cited by 1 publication

References 22 publications

Lessons from the ATTeST trial in ataxia telangiectasia

Lessons from the ATTeST trial in ataxia telangiectasia

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