Title |
Intra-Erythrocyte Infusion of Dexamethasone Reduces Neurological Symptoms in Ataxia Teleangiectasia Patients: Results of a Phase 2 Trial
|
---|---|
Published in |
Orphanet Journal of Rare Diseases, January 2014
|
DOI | 10.1186/1750-1172-9-5 |
Pubmed ID | |
Authors |
Luciana Chessa, Vincenzo Leuzzi, Alessandro Plebani, Annarosa Soresina, Roberto Micheli, Daniela D’Agnano, Tullia Venturi, Anna Molinaro, Elisa Fazzi, Mirella Marini, Pierino Ferremi Leali, Isabella Quinti, Filomena Monica Cavaliere, Gabriella Girelli, Maria Cristina Pietrogrande, Andrea Finocchi, Stefano Tabolli, Damiano Abeni, Mauro Magnani |
Abstract |
Ataxia Teleangiectasia [AT] is a rare neurodegenerative disease characterized by early onset ataxia, oculocutaneous teleangiectasias, immunodeficiency, recurrent infections, radiosensitivity and proneness to cancer. No therapies are available for this devastating disease. Recent observational studies in few patients showed beneficial effects of short term treatment with betamethasone. To avoid the characteristic side effects of long-term administration of steroids we developed a method for encapsulation of dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) allowing slow release of dexamethasone for up to one month after dosing. Aims of the study were: the assessment of the effect of EryDex in improving neurological symptoms and adaptive behaviour of AT patients; the safety and tolerability of the therapy. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
Mexico | 1 | 50% |
Unknown | 1 | 50% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 2 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Poland | 1 | 1% |
Unknown | 91 | 99% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 17 | 18% |
Student > Master | 13 | 14% |
Researcher | 12 | 13% |
Student > Bachelor | 12 | 13% |
Student > Doctoral Student | 6 | 7% |
Other | 17 | 18% |
Unknown | 15 | 16% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 17 | 18% |
Agricultural and Biological Sciences | 10 | 11% |
Biochemistry, Genetics and Molecular Biology | 9 | 10% |
Chemistry | 7 | 8% |
Nursing and Health Professions | 6 | 7% |
Other | 21 | 23% |
Unknown | 22 | 24% |