Data sharing to advance gene-targeted therapies in rare diseases.

Lekstrom-Himes, Julie; Augustine, Erika F; Brower, Amy; Defay, Thomas; Finkel, Richard S; McGuire, Amy L; Skinner, Mark W; Yu, Timothy W

Lekstrom-Himes, Julie; Augustine, Erika F; Brower, Amy; Defay, Thomas; Finkel, Richard S; McGuire, Amy L; Skinner, Mark W; Yu, Timothy W.

Afiliación

Lekstrom-Himes J; Takeda, Cambridge, Massachusetts, USA.
Augustine EF; Department of Neurology and Developmental Medicine, Kennedy Krieger Institute, Baltimore, Maryland, USA.
Brower A; American College of Medical Genetics and Genomics, Bethesda, Maryland, USA.
Defay T; Alexion Pharmaceuticals, Boston, Massachusetts, USA.
Finkel RS; Center for Experimental Neurotherapeutics, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
McGuire AL; Center for Medical Ethics and Health Policy, Baylor College of Medicine, Houston, Texas, USA.
Skinner MW; Institute for Policy Advancement Ltd., Washington, DC, USA.
Yu TW; Department of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, Ontario, Canada.

Am J Med Genet C Semin Med Genet ; 193(1): 87-98, 2023 03.

Article en En | MEDLINE | ID: mdl-36594517

Asunto(s)

Difusión de la Información; Enfermedades Raras; Humanos; Enfermedades Raras/genética; Enfermedades Raras/terapia

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedades Raras / Difusión de la Información Tipo de estudio: Screening_studies Límite: Humans Idioma: En Revista: Am J Med Genet C Semin Med Genet Asunto de la revista: GENETICA MEDICA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos

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