. "Voretigene neparvovec es un medicamento que se utiliza para el tratamiento de la amaurosis cong\u00E9nita de Leber. No cura la enfermedad, pero puede mejorar la visi\u00F3n de las personas afectadas. Se administra mediante inyecci\u00F3n subrretiniana. Se vende con el nombre comercial de Luxturna. Es una terapia g\u00E9nica. Su empleo fue aprobado por la Administraci\u00F3n de Alimentos y Medicamentos de Estados Unidos (FDA) en marzo de 2018.\u200B \u200B \u200B La amaurosis cong\u00E9nita de Leber es una enfermedad de la retina (retinopat\u00EDa) de origen gen\u00E9tico, provoca d\u00E9ficit visual en los ni\u00F1os afectados desde los primeros meses de vida. Supone entre el 10-18% de los casos de ceguera cong\u00E9nita y su incidencia es de 1 de cada 35 000 nacidos vivos. Fue descrita por el oftalm\u00F3logo Theodor Leber en el siglo XIX."@es . . "Voretigene neparvovec es un medicamento que se utiliza para el tratamiento de la amaurosis cong\u00E9nita de Leber. No cura la enfermedad, pero puede mejorar la visi\u00F3n de las personas afectadas. Se administra mediante inyecci\u00F3n subrretiniana. Se vende con el nombre comercial de Luxturna. Es una terapia g\u00E9nica. Su empleo fue aprobado por la Administraci\u00F3n de Alimentos y Medicamentos de Estados Unidos (FDA) en marzo de 2018.\u200B \u200B \u200B La amaurosis cong\u00E9nita de Leber es una enfermedad de la retina (retinopat\u00EDa) de origen gen\u00E9tico, provoca d\u00E9ficit visual en los ni\u00F1os afectados desde los primeros meses de vida. Supone entre el 10-18% de los casos de ceguera cong\u00E9nita y su incidencia es de 1 de cada 35 000 nacidos vivos. Fue descrita por el oftalm\u00F3logo Theodor Leber en el siglo XIX."@es . "55488052"^^ . "B2"@en . "DB13932" . "2SPI046IKD" . . . . . "S01"@en . . . "1124269887"^^ . . . "Rx-only / Schedule D"@en . . "Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis. Leber's congenital amaurosis, or biallelic RPE65-mediated inherited retinal disease, is an inherited disorder causing progressive blindness. Voretigene is the first treatment available for this condition. The gene therapy is not a cure for the condition, but substantially improves vision in those treated. It is given as a subretinal injection. Voretigene neparvovec was approved for medical use in the United States in December 2017, Australia in August 2020 and in Canada, in October 2020. It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA)."@en . . . "yes"@en . . "1646819"^^ . . . . "S4"@en . "\u0645\u0633\u062A\u062D\u0636\u0631 \u0644\u0648\u0643\u0633\u062A\u0648\u0631\u0646\u0627 (\u0641\u0648\u0631\u062A\u064A\u062C\u064A\u0646 \u0646\u064A\u0628\u0627\u0631\u0648\u0641\u0648\u0641\u064A\u0643) \u0647\u0648 \u0639\u0644\u0627\u062C \u062C\u064A\u0646\u064A \u062C\u062F\u064A\u062F \u0644\u0639\u0644\u0627\u062C \u0641\u0642\u062F\u0627\u0646 \u0627\u0644\u0628\u0635\u0631 \u0627\u0644\u0648\u0631\u0627\u062B\u064A \u0627\u0644\u0630\u064A \u0642\u062F \u064A\u0624\u062F\u064A \u0625\u0644\u0649 \u0627\u0644\u0639\u0645\u0649 . \u062A\u0645 \u062A\u0637\u0648\u064A\u0631\u0647 \u0645\u0646 \u0642\u0628\u0644 Spark Therapeutics \u0648\u0645\u0633\u062A\u0634\u0641\u0649 \u0627\u0644\u0623\u0637\u0641\u0627\u0644 \u0641\u064A \u0641\u064A\u0644\u0627\u062F\u0644\u0641\u064A\u0627 . \u0647\u0630\u0627 \u0647\u0648 \u0627\u0644\u0639\u0644\u0627\u062C \u0627\u0644\u062C\u064A\u0646\u064A \u0627\u0644\u0623\u0648\u0644 \u0627\u0644\u0630\u064A \u0648\u0627\u0641\u0642\u062A \u0639\u0644\u064A\u0647 \u0625\u062F\u0627\u0631\u0629 \u0627\u0644\u0623\u063A\u0630\u064A\u0629 \u0648\u0627\u0644\u0639\u0642\u0627\u0642\u064A\u0631. \u0648\u064A\u0639\u062F \u0644\u0648\u0643\u0633\u062A\u0648\u0631\u0646\u0627 \u0623\u0648\u0644 \u0639\u0644\u0627\u062C \u062C\u064A\u0646\u064A \u064A\u0633\u062A\u0647\u062F\u0641 \u0645\u0631\u0636\u0627\u064B \u0646\u0627\u062C\u0645\u0627\u064B \u0639\u0646 \u0637\u0641\u0631\u0627\u062A \u0641\u064A \u062C\u064A\u0646 \u0645\u062D\u062F\u062F\u060C \u0648\u062A\u0645\u062A \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0639\u0644\u0649 \u0644\u0648\u0643\u0633\u062A\u0648\u0631\u0646\u0627 \u0644\u0639\u0644\u0627\u062C \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0628\u0627\u0644\u063A\u064A\u0646 \u0648\u0627\u0644\u0623\u0637\u0641\u0627\u0644 \u0627\u0644\u0630\u064A\u0646 \u064A\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u0641\u0642\u062F\u0627\u0646 \u0627\u0644\u0628\u0635\u0631 \u0628\u0633\u0628\u0628 \u062D\u062B\u0644 \u0627\u0644\u0634\u0628\u0643\u064A\u0629 \u0627\u0644\u0648\u0631\u0627\u062B\u064A \u0627\u0644\u0646\u0627\u062C\u0645 \u0639\u0646 \u062D\u062F\u0648\u062B \u0637\u0641\u0631\u0627\u062A \u0641\u064A \u062C\u064A\u0646 (RPE65)\u060C \u0625\u0630 \u062A\u0645\u0646\u0639 \u0647\u0630\u0647 \u0627\u0644\u0637\u0641\u0631\u0627\u062A \u0627\u0644\u062C\u0633\u0645 \u0645\u0646 \u0625\u0646\u062A\u0627\u062C \u0623\u062D\u062F \u0627\u0644\u0628\u0631\u0648\u062A\u064A\u0646\u0627\u062A \u0627\u0644\u0636\u0631\u0648\u0631\u064A\u0629 \u0644\u0644\u0631\u0624\u064A\u0629 \u0645\u0627 \u064A\u0624\u062F\u064A \u0625\u0644\u0649 \u0641\u0642\u062F\u0627\u0646 \u0627\u0644\u0628\u0635\u0631 \u0648\u0627\u0644\u0639\u0645\u0649 \u0641\u064A \u0646\u0647\u0627\u064A\u0629 \u0627\u0644\u0645\u0637\u0627\u0641"@ar . . "Rx-only"@en . . . . "D11008"@en . . "1646819-03-5" . "Rx-only"@en . . . . "\u0645\u0633\u062A\u062D\u0636\u0631 \u0644\u0648\u0643\u0633\u062A\u0648\u0631\u0646\u0627 (\u0641\u0648\u0631\u062A\u064A\u062C\u064A\u0646 \u0646\u064A\u0628\u0627\u0631\u0648\u0641\u0648\u0641\u064A\u0643)"@ar . . . . . "Luxturna"@en . . . . . . "Luxturna"@en . "Adeno-associated virus serotype 2"@en . . "D11008" . . "Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis. Leber's congenital amaurosis, or biallelic RPE65-mediated inherited retinal disease, is an inherited disorder causing progressive blindness. Voretigene is the first treatment available for this condition. The gene therapy is not a cure for the condition, but substantially improves vision in those treated. It is given as a subretinal injection."@en . "XA27"@en . . . . "voretigene neparvovec-rzyl"@en . . "Luxturna"@en . . . "2"^^ . . . "Voretigene neparvovec"@es . "Voretigene_neparvovec"@en . . "\u0645\u0633\u062A\u062D\u0636\u0631 \u0644\u0648\u0643\u0633\u062A\u0648\u0631\u0646\u0627 (\u0641\u0648\u0631\u062A\u064A\u062C\u064A\u0646 \u0646\u064A\u0628\u0627\u0631\u0648\u0641\u0648\u0641\u064A\u0643) \u0647\u0648 \u0639\u0644\u0627\u062C \u062C\u064A\u0646\u064A \u062C\u062F\u064A\u062F \u0644\u0639\u0644\u0627\u062C \u0641\u0642\u062F\u0627\u0646 \u0627\u0644\u0628\u0635\u0631 \u0627\u0644\u0648\u0631\u0627\u062B\u064A \u0627\u0644\u0630\u064A \u0642\u062F \u064A\u0624\u062F\u064A \u0625\u0644\u0649 \u0627\u0644\u0639\u0645\u0649 . \u062A\u0645 \u062A\u0637\u0648\u064A\u0631\u0647 \u0645\u0646 \u0642\u0628\u0644 Spark Therapeutics \u0648\u0645\u0633\u062A\u0634\u0641\u0649 \u0627\u0644\u0623\u0637\u0641\u0627\u0644 \u0641\u064A \u0641\u064A\u0644\u0627\u062F\u0644\u0641\u064A\u0627 . \u0647\u0630\u0627 \u0647\u0648 \u0627\u0644\u0639\u0644\u0627\u062C \u0627\u0644\u062C\u064A\u0646\u064A \u0627\u0644\u0623\u0648\u0644 \u0627\u0644\u0630\u064A \u0648\u0627\u0641\u0642\u062A \u0639\u0644\u064A\u0647 \u0625\u062F\u0627\u0631\u0629 \u0627\u0644\u0623\u063A\u0630\u064A\u0629 \u0648\u0627\u0644\u0639\u0642\u0627\u0642\u064A\u0631. \u0648\u064A\u0639\u062F \u0644\u0648\u0643\u0633\u062A\u0648\u0631\u0646\u0627 \u0623\u0648\u0644 \u0639\u0644\u0627\u062C \u062C\u064A\u0646\u064A \u064A\u0633\u062A\u0647\u062F\u0641 \u0645\u0631\u0636\u0627\u064B \u0646\u0627\u062C\u0645\u0627\u064B \u0639\u0646 \u0637\u0641\u0631\u0627\u062A \u0641\u064A \u062C\u064A\u0646 \u0645\u062D\u062F\u062F\u060C \u0648\u062A\u0645\u062A \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0639\u0644\u0649 \u0644\u0648\u0643\u0633\u062A\u0648\u0631\u0646\u0627 \u0644\u0639\u0644\u0627\u062C \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0628\u0627\u0644\u063A\u064A\u0646 \u0648\u0627\u0644\u0623\u0637\u0641\u0627\u0644 \u0627\u0644\u0630\u064A\u0646 \u064A\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u0641\u0642\u062F\u0627\u0646 \u0627\u0644\u0628\u0635\u0631 \u0628\u0633\u0628\u0628 \u062D\u062B\u0644 \u0627\u0644\u0634\u0628\u0643\u064A\u0629 \u0627\u0644\u0648\u0631\u0627\u062B\u064A \u0627\u0644\u0646\u0627\u062C\u0645 \u0639\u0646 \u062D\u062F\u0648\u062B \u0637\u0641\u0631\u0627\u062A \u0641\u064A \u062C\u064A\u0646 (RPE65)\u060C \u0625\u0630 \u062A\u0645\u0646\u0639 \u0647\u0630\u0647 \u0627\u0644\u0637\u0641\u0631\u0627\u062A \u0627\u0644\u062C\u0633\u0645 \u0645\u0646 \u0625\u0646\u062A\u0627\u062C \u0623\u062D\u062F \u0627\u0644\u0628\u0631\u0648\u062A\u064A\u0646\u0627\u062A \u0627\u0644\u0636\u0631\u0648\u0631\u064A\u0629 \u0644\u0644\u0631\u0624\u064A\u0629 \u0645\u0627 \u064A\u0624\u062F\u064A \u0625\u0644\u0649 \u0641\u0642\u062F\u0627\u0646 \u0627\u0644\u0628\u0635\u0631 \u0648\u0627\u0644\u0639\u0645\u0649 \u0641\u064A \u0646\u0647\u0627\u064A\u0629 \u0627\u0644\u0645\u0637\u0627\u0641"@ar . . . "14433"^^ . . . . . . . . "Voretigene neparvovec"@en . "DB13932"@en .