Study methodology and insights from the palovarotene clinical development program in fibrodysplasia ossificans progressiva

BACKGROUND: The design of clinical trials in rare diseases is often complicated by a lack of real-world translational knowledge. Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by skeletal malformations and progressive heterotopic ossification (HO). Palova...

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Detalles Bibliográficos
Autores principales: Pignolo, Robert J., Al Mukaddam, Mona, Baujat, Geneviève, Brown, Matthew A., De Cunto, Carmen, Hsiao, Edward C., Keen, Richard, Le Quan Sang, Kim-Hanh, Grogan, Donna R., Marino, Rose, Strahs, Andrew R., Kaplan, Frederick S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10642058/
https://www.ncbi.nlm.nih.gov/pubmed/37957586
http://dx.doi.org/10.1186/s12874-023-02080-7

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