Intra-erythrocyte infusion of dexamethasone reduces neurological symptoms in ataxia teleangiectasia patients: Results of a phase 2 trial

Orphanet Journal of Rare Diseases

Volumn 9, Issue 1, 2014, Pages

  Chessa, Luciana ^a   Leuzzi, Vincenzo ^a   Plebani, Alessandro ^b   Soresina, Annarosa ^b   Micheli, Roberto ^b   D'Agnano, Daniela ^a   Venturi, Tullia ^a   Molinaro, Anna ^c   Fazzi, Elisa ^b   Marini, Mirella ^b   Ferremi Leali, Pierino ^b   Quinti, Isabella ^d   Cavaliere, Filomena Monica ^d   Girelli, Gabriella ^d   Pietrogrande, Maria Cristina ^e   Finocchi, Andrea ^f   Tabolli, Stefano ^g   Abeni, Damiano ^g   Magnani, Mauro ^h  

^a SAPIENZA UNIVERSITY OF ROME   (Italy)

^b UNIVERSITY OF BRESCIA   (Italy)

^c UNIVERSITY OF TRIESTE   (Italy)

^d SAPIENZA UNIVERSITY OF ROME   (Italy)

^e UNIVERSITY OF MILAN   (Italy)

^f UNIVERSITY OF ROME TOR VERGATA   (Italy)

^g LABORATORIO DI PATOLOGIA VASCOLARE   (Italy)

^h UNIVERSITY OF URBINO   (Italy)

Author keywords

Ataxia Teleangiectasia; Ataxia Teleangiectasia Ataxia Treatment; Cerebellar Ataxia; Dexamethasone; ICARS; Intra Erythrocyte Dexamethasone; VABS

Indexed keywords

ADOLESCENT; ATAXIA TELANGIECTASIA; CHILD; DEXAMETHASONE; DISEASE PROGRESSION; ERYTHROCYTES; FEMALE; HUMANS; MALE;

EID: 84892158748     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-9-5     Document Type: Article

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