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FDA Sets Review Date for Gene Therapy for Enzyme Deficiency.

Subjects: GENE therapy; RARE diseases; ENZYMESUNITED KingdomUNITED States. Food & Drug Administration

• Source: Formulary Watch. 5/14/2024, p1-2. 2p.

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Pondering the Promise Of Gene Therapy.

• Authors : Smith, Gerry; Contiliano, Tom

Subjects: *HEALTH facilities; GENE therapy; SPOKESPERSONSBIOMARIN Pharmaceutical Inc. BIOMARIN Pharmaceutical Inc. BIOMARIN Pharmaceutical Inc. BMRN

• Source: Bloomberg Businessweek. 2/26/2024, Issue 4813, p10-12. 3p. 1 Graph, 1 Cartoon or Caricature.

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Extension study of novel topical gene therapy reveals positive results.

• Authors : Wehrwein, Peter

Subjects: GENE therapy; SKIN diseases

• Source: Managed Healthcare Executive. Apr2024, Vol. 34 Issue 4, p40-41. 2p.

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The Gene Fix.

• Authors : Landhuis, Esther

Subjects: GENE therapy; HEMATOLOGIC malignancies; MUSCLE diseases

• Source: Scientific American. Nov2021, Vol. 325 Issue 5, pS3-S5. 3p. 1 Diagram.

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Gene Therapy Arrives.

• Authors : Daley, Jim

Subjects: GENE therapy; GENETIC mutation; LIPOPROTEIN lipaseEUROPEAN Union

• Source: Scientific American. Jan2020, Vol. 322 Issue 1, pS12-S13. 2p. 1 Color Photograph.

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Pfizer Pauses Gene Therapy Trial for Muscle Disease After Death.

• Authors : Garde, Damian (AUTHOR)

Subjects: GENE therapy; MUSCLE diseases; CARDIAC patientsPFIZER Inc. 001326495 PFIZER Inc. PFIZER Inc. 001326495 001326495 PFIZER Inc. 001326495 PFE

• Source: Bloomberg.com. 5/7/2024, pN.PAG-N.PAG. 1p.

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Success Stories.

• Authors : Daley, Jim

Subjects: GENE therapy; NEUROMUSCULAR diseases; BLINDNESS

• Source: Scientific American. Nov2021, Vol. 325 Issue 5, pS12-S14. 3p.

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Editing the Book of Life.

• Authors : E. L.

Subjects: GENE therapy; BONE marrow

• Source: Scientific American. Nov2021, Vol. 325 Issue 5, pS6-S7. 2p. 1 Diagram.

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Are value-based contracts the answer to ultra-high-priced gene therapies?

• Authors : Myshko, Denise

Subjects: GENE therapy; SICKLE cell anemiaUNITED States. Food & Drug Administration

• Source: Managed Healthcare Executive. May2024, Vol. 34 Issue 5, p66-67. 2p.

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Gene therapy proves successful in treating hereditary deafness.

• Authors : Reisinger, Ellen¹ (AUTHOR); Trapani, Ivana^2,3 (AUTHOR)

Subjects: GENE therapy; DEAFNESS

• Source: Lancet. May2024, Vol. 403 Issue 10441, p2267-2269. 3p.

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