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Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome

BACKGROUND: Natural history studies are increasingly recognized as having an important role in drug development for rare diseases. A phase 3, observational, retrospective, and non-interventional study was designed to establish a natural history control (NHC) cohort of patients with Barth syndrome (B...

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Detalles Bibliográficos
Autores principales:	Hornby, Brittany, Thompson, William Reid, Almuqbil, Mohammed, Manuel, Ryan, Abbruscato, Anthony, Carr, Jim, Vernon, Hilary J.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9438322/ https://www.ncbi.nlm.nih.gov/pubmed/36056411 http://dx.doi.org/10.1186/s13023-022-02469-5

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