Precision Medicines’ Impact on Orphan Drug Designation
Mueller, Christine M., Rao, Gayatri R., Miller Needleman, Katherine I.
Published in Clinical and translational science (01.11.2019)
Published in Clinical and translational science (01.11.2019)
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Journal Article
Flexibility in the FDA approach to orphan drug development
Hunter, Nina L, Rao, Gayatri R, Sherman, Rachel E
Published in Nature reviews. Drug discovery (01.11.2017)
Published in Nature reviews. Drug discovery (01.11.2017)
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Journal Article
Children and Adults With Rare Diseases Need Innovative Medical Devices
Peiris, Vasum, Xu, Kui, Agler, Heather L, Chen, Eric A, Gopal-Srivastava, Rashmi, Lappin, Brian M, Lewis, Debra Y, Rao, Gayatri R
Published in Journal of medical devices (01.09.2018)
Published in Journal of medical devices (01.09.2018)
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Journal Article
Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials
Czechowicz, Agnieszka, Sevilla, Julián, Booth, Claire, Agarwal, Rajni, Zubicaray, Josune, Río, Paula, Navarro, Susana, Chetty, Kritika, O'Toole, Grainne, Xu-Bayford, Jinhua, Ancliff, Philip, Sebastián, Elena, Choi, Grace, Zeini, Miriam, Nicoletti, Eileen, Wagner, John E, Rao, Gayatri R., Thrasher, Adrian J., Schwartz, Jonathan D., Roncarolo, Maria Grazia, Bueren, Juan A.
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
Booth, Claire, Sevilla, Julián, Rao, Gayatri R., Chitty Lopez, Maria, Almarza, Elena, Terrazas, Dayna, Zubicaray, Josune, González Vicent, Marta, Chetty, Kritika, O'Toole, Grainne, Xu-Bayford, Jinhua, Nicoletti, Eileen, Fernandes, Augustine, Kuo, Caroline, de Oliveira, Satiro, Moore, Theodore B., Choi, Grace, Zeini, Miriam, Mesa-Núñez, Cristina, Thrasher, Adrian J., Bueren, Juan A., Schwartz, Jonathan D., Kohn, Donald B.
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study
Shah, Ami J., López Lorenzo, José Luis, Sevilla, Julián, Navarro, Susana, Llanos, Lucía, Pérez de Camino Gaisse, Begoña, Sanchez, Sol, Zubicaray, Josune, Glader, Bert, Chien, May, Quintana Bustamante, Oscar, Zeini, Miriam, Choi, Grace, Nicoletti, Eileen, Rao, Gayatri R., Roncarolo, Maria Grazia, Bueren, Juan A., Schwartz, Jonathan D., Segovia, José C.
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results
Kohn, Donald B., Booth, Claire, Sevilla, Julián, Rao, Gayatri R, Almarza, Elena, Terrazas, Dayna, Nicoletti, Eileen, Fernandes, Augustine, Kuo, Caroline, de Oliveira, Satiro, Moore, Theodore B., Law, Kenneth M, Beard, Brian C, Choi, Grace, Zeini, Miriam, Mesa-Núñez, Cristina, Thrasher, Adrian J., Bueren, Juan A, Schwartz, Jonathan D
Published in Blood (23.11.2021)
Published in Blood (23.11.2021)
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Journal Article
Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials
Czechowicz, Agnieszka, Sevilla, Julián, Agarwal, Rajni, Booth, Claire, Zubicaray, Josune, Río, Paula, Navarro, Susana, Ancliff, Philip, Sebastian, Elena, Beard, Brian C, Law, Kenneth M, Choi, Grace, Zeini, Miriam, Duran-Persson, Camilla, Nicoletti, Eileen, Wagner, John E, Rao, Gayatri R, Thrasher, Adrian J., Schwartz, Jonathan D, Bueren, Juan A, Roncarolo, Maria Grazia
Published in Blood (23.11.2021)
Published in Blood (23.11.2021)
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Journal Article
Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients
Shah, Ami J, López Lorenzo, José Luis, Navarro, Susana, Sevilla, Julián, Llanos, Lucía, Pérez de Camino Gaisse, Begoña, Sanchez, Sol, Glader, Bert, Chien, May, Quintana Bustamante, Oscar, Beard, Brian C, Law, Kenneth M, Zeini, Miriam, Choi, Grace, Nicoletti, Eileen, Rao, Gayatri R, Roncarolo, Maria Grazia, Bueren, Juan A, Schwartz, Jonathan D, Segovia, José C
Published in Blood (23.11.2021)
Published in Blood (23.11.2021)
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Journal Article
Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients
López Lorenzo, José Luis, Navarro, Susana, Shah, Ami J, Roncarolo, Maria Grazia, Sevilla, Julián, Llanos, Lucía, Pérez Camino de Gaisse, Begoña, Sanchez, Sol, Glader, Bertil, Chien, May, Quintana Bustamante, Oscar, Beard, Brian C, Law, Kenneth M, Zeini, Miriam, Choi, Grace, Nicoletti, Eileen, Bueren, Juan A, Rao, Gayatri R, Schwartz, Jonathan D, Segovia, José C
Published in Blood (05.11.2020)
Published in Blood (05.11.2020)
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Journal Article