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Protocol paper: a multi-center, double-blinded, randomized, 6-month, placebo-controlled study followed by 12-month open label extension to evaluate the safety and efficacy of Saracatinib in Fibrodysplasia Ossificans Progressiva (STOPFOP)
BACKGROUND: Fibrodysplasia Ossificans Progressiva (FOP) is a genetic, progressive and devastating disease characterized by severe heterotopic ossification (HO), loss of mobility and early death. There are no FDA approved medications. The STOPFOP team identified AZD0530 (saracatinib) as a potent inhi...
Autores principales: | Smilde, Bernard J., Stockklausner, Clemens, Keen, Richard, Whittaker, Andrew, Bullock, Alex N., von Delft, Annette, van Schoor, Natasja M., Yu, Paul B., Eekhoff, E. Marelise W. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9156821/ https://www.ncbi.nlm.nih.gov/pubmed/35650602 http://dx.doi.org/10.1186/s12891-022-05471-x |
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