Concepedia

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, Mauro Magnani, Matthias Kieslich, Monique M. Ryan, Isabelle Meyts, Sheffali Gulati, Rupam Borgohain, Ravi Yadav,

The Lancet Neurology · 2024 · 25 citations · 17 references

DOIFull text

Open access

Concepts

References

17

International Cooperative Ataxia Rating Scale for pharmacological assessment of the cerebellar syndrome

P Trouillas, T Takayanagi, Mark Hallett et al. · Journal of the Neurological Sciences · 1997 · 1.3K citations

, , +15

Intra-Erythrocyte Infusion of Dexamethasone Reduces Neurological Symptoms in Ataxia Teleangiectasia Patients: Results of a Phase 2 Trial

Luciana Chessa, Vincenzo Leuzzi, Alessandro Plebani et al. · Orphanet Journal of Rare Diseases · 2014 · 142 citations · Full text

, , +11

Accumulation of DNA Damage and Reduced Levels of Nicotine Adenine Dinucleotide in the Brains of Atm-deficient Mice

Nora Stern, Ayala Hochman, Naty Zemach et al. · Journal of Biological Chemistry · 2002 · 104 citations · Full text

, , +17

Inhibition of the cGAS‐STING pathway ameliorates the premature senescence hallmarks of Ataxia‐Telangiectasia brain organoids

Julio Aguado, Harman Kaur Chaggar, Cecilia Gómez‐Inclán et al. · Aging Cell · 2021 · 91 citations · Full text

, , +17

A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia

Raffaella Zannolli, Sabrina Buoni, Gianni Betti et al. · Movement Disorders · 2012 · 91 citations

, , +8