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Biomarker Identification, Safety, and Efficacy of High-Dose Antioxidants for Adrenomyeloneuropathy: a Phase II Pilot Study
X-Adrenoleukodystrophy (X-ALD) and its adult-onset, most prevalent variant adrenomyeloneuropathy (AMN) are caused by mutations in the peroxisomal transporter of the very long-chain fatty acid ABCD1. AMN patients classically present spastic paraparesis that can progress over decades, and a satisfacto...
Autores principales: | Casasnovas, Carlos, Ruiz, Montserrat, Schlüter, Agatha, Naudí, Alba, Fourcade, Stéphane, Veciana, Misericordia, Castañer, Sara, Albertí, Antonia, Bargalló, Nuria, Johnson, Maria, Raymond, Gerald V., Fatemi, Ali, Moser, Ann B., Villarroya, Francesc, Portero-Otín, Manuel, Artuch, Rafael, Pamplona, Reinald, Pujol, Aurora |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer International Publishing
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6985062/ https://www.ncbi.nlm.nih.gov/pubmed/31077039 http://dx.doi.org/10.1007/s13311-019-00735-2 |
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