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17 results on '"Ouyang, Haojun"'

1. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Author

Seferian, A., De Lucia, S., Tachibana, S., Jollet, A., Mouffak, S., Pedemonte, M., Brolatti, N., Morando, S., Vanlander, A., De Vos, E., Tahon, V., Govoni, A., Magri, F., Comi, G., Foa, M., Parente, V., Buscemi, L., Dal Farra, F., Schneider, O., Jonas, A., Defeldre, A.C., Pagliano, E., Zanin, R., Arnoldi, M.T., Schembri, V., Del Sole, M., Mandelli, A., Pera, M.C., Antonaci, L., Coratti, G., de Sanctis, R., Pane, M., Scoto, M., Groves, K., Edel, L., Abel, F., Van Ruiten, H., Lofra, R.M., Thompson, E., Mercuri, Eugenio, Muntoni, Francesco, Baranello, Giovanni, Masson, Riccardo, Boespflug-Tanguy, Odile, Bruno, Claudio, Corti, Stefania, Daron, Aurore, Deconinck, Nicolas, Servais, Laurent, Straub, Volker, Ouyang, Haojun, Chand, Deepa, Tauscher-Wisniewski, Sitra, Mendonca, Nuno, and Lavrov, Arseniy

Published
2021
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2. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

Author

Day, John W, Finkel, Richard S, Chiriboga, Claudia A, Connolly, Anne M, Crawford, Thomas O, Darras, Basil T, Iannaccone, Susan T, Kuntz, Nancy L, Peña, Loren D M, Shieh, Perry B, Smith, Edward C, Kwon, Jennifer M, Zaidman, Craig M, Schultz, Meredith, Feltner, Douglas E, Tauscher-Wisniewski, Sitra, Ouyang, Haojun, Chand, Deepa H, Sproule, Douglas M, Macek, Thomas A, and Mendell, Jerry R

Published
2021
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4. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Author

Mercuri, Eugenio, primary, Muntoni, Francesco, additional, Baranello, Giovanni, additional, Masson, Riccardo, additional, Boespflug-Tanguy, Odile, additional, Bruno, Claudio, additional, Corti, Stefania, additional, Daron, Aurore, additional, Deconinck, Nicolas, additional, Servais, Laurent, additional, Straub, Volker, additional, Ouyang, Haojun, additional, Chand, Deepa, additional, Tauscher-Wisniewski, Sitra, additional, Mendonca, Nuno, additional, Lavrov, Arseniy, additional, Seferian, A., additional, De Lucia, S., additional, Tachibana, S., additional, Jollet, A., additional, Mouffak, S., additional, Pedemonte, M., additional, Brolatti, N., additional, Morando, S., additional, Vanlander, A., additional, De Vos, E., additional, Tahon, V., additional, Govoni, A., additional, Magri, F., additional, Comi, G., additional, Foa, M., additional, Parente, V., additional, Buscemi, L., additional, Dal Farra, F., additional, Schneider, O., additional, Jonas, A., additional, Defeldre, A.C., additional, Pagliano, E., additional, Zanin, R., additional, Arnoldi, M.T., additional, Schembri, V., additional, Del Sole, M., additional, Mandelli, A., additional, Pera, M.C., additional, Antonaci, L., additional, Coratti, G., additional, de Sanctis, R., additional, Pane, M., additional, Scoto, M., additional, Groves, K., additional, Edel, L., additional, Abel, F., additional, Van Ruiten, H., additional, Lofra, R.M., additional, and Thompson, E., additional

Published
2021
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6. Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update (1828)

Author

Day, John W., primary, Chiriboga, Claudia A., additional, Crawford, Thomas O., additional, Darras, Basil T., additional, Finkel, Richard S., additional, Connolly, Anne M., additional, Iannaccone, Susan T., additional, Kuntz, Nancy L., additional, Peña, Loren D.M., additional, Shieh, Perry B., additional, Smith, Edward C., additional, Schultz, Meredith, additional, Feltner, Douglas E., additional, Tauscher-Wisniewski, Sitra, additional, Ogrinc, Francis G., additional, Shah, Ankita, additional, Ouyang, Haojun, additional, Macek, Thomas, additional, Kernbauer, Elaine, additional, Sproule, Douglas M., additional, and Mendell, Jerry R., additional

Published
2020
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7. Gene-Replacement Therapy in Spinal Muscular Atrophy Type 1: Long-Term Follow-Up From the Onasemnogene Abeparvovec-xioi Phase 1/2a Clinical Trial (1808)

Author

Mendell, Jerry R., primary, Shell, Richard, additional, Lehman, Kelly J., additional, McColly, Markus, additional, Lowes, Linda P., additional, Alfano, Lindsay N., additional, Miller, Natalie F., additional, Iammarino, Megan A., additional, Church, Kathleen, additional, Ogrinc, Francis G., additional, Ouyang, Haojun, additional, Kernbauer, Elaine, additional, Joshi, Shivani, additional, Sproule, Douglas M., additional, Meriggioli, Matthew, additional, Feltner, Douglas E., additional, and Al-Zaidy, Samiah, additional

Published
2020
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8. Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update

Author

Day, John W., additional, Chiriboga, Claudia A., additional, Crawford, Thomas O., additional, Darras, Basil T., additional, Finkel, Richard S., additional, Connolly, Anne M., additional, Iannaccone, Susan T., additional, Kuntz, Nancy L., additional, Peña, Loren, additional, Schultz, Meredith, additional, Shieh, Perry B., additional, Smith, Edward C., additional, Ernst, Uwe, additional, Feltner, Douglas E., additional, Orgrinc, Francis G., additional, Shah, Ankita, additional, Ouyang, Haojun, additional, Macek, Thomas A., additional, Kernbauer, Elaine, additional, L’Italien, James, additional, Sproule, Douglas M., additional, Kaspar, Brian K., additional, and Medel, Jerry R., additional

Published
2019
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9. Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe

Author

Muntoni, Francesco, additional, Baranello, Giovanni, additional, Bruno, Claudio, additional, Corti, Stefania, additional, Masson, Riccardo, additional, Straub, Volker, additional, Vita, Giuseppe, additional, Ernst, Uwe, additional, Kernbauer, Elaine, additional, Williamson, Susan, additional, Ouyang, Haojun, additional, Feltner, Douglas E., additional, Meriggioli, Matthew, additional, Lavrov, Arseniy, additional, L’Italien, James, additional, Sproule, Douglas M., additional, and Mercuri, Eugenio, additional

Published
2019
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10. Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial

Author

Mendell, Jerry R., additional, Shell, Richard, additional, Lehman, Kelly J., additional, McColly, Markus, additional, Lowes, Linda P., additional, Alfano, Lindsay N., additional, Miller, Natalie F., additional, Iammarino, Megan A., additional, Church, Kathleen, additional, Ernst, Uwe, additional, Ogrinc, Francis G., additional, Ouyang, Haojun, additional, Kernbauer, Elaine, additional, Shah, Shivani, additional, L’Italien, James, additional, Sproule, Douglas M., additional, Feltner, Douglas E., additional, and Al-Zaidy, Samiah, additional

Published
2019
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11. Establishing Clinical Trial Readiness of the Rett Syndrome Hand Function Measure (P1.6-034)

Author

Leonard, Helen, primary, Benke, Timothy, additional, Diener, Pamela S., additional, Feltner, Douglas, additional, Kaufmann, Petra, additional, Lieberman, David N., additional, LoCicero, Amie, additional, Macek, Thomas A., additional, McGill, Bryan E., additional, Miner, Julie K., additional, Muehring, Lynlee, additional, Neul, Jeffrey L., additional, O’Keefe, Joan A., additional, Ouyang, Haojun, additional, Percy, Alan, additional, Tarquinio, Daniel C., additional, and Downs, Jenny, additional

Published
2019
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12. Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial.

Author

Mendell, Jerry R., Shell, Richard, Lehman, Kelly J., McColly, Markus, Lowes, Linda P., Alfano, Lindsay N., Miller, Natalie F., Iammarino, Megan A., Church, Kathleen, Ernst, Uwe, Ogrinc, Francis G., Ouyang, Haojun, Kernbauer, Elaine, Shah, Shivani, L'Italien, James, Sproule, Douglas M., Feltner, Douglas E., and Al-Zaidy, Samiah

Subjects
SPINAL muscular atrophy, CLINICAL trials
Published
2019
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13. Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe.

Author

Muntoni, Francesco, Baranello, Giovanni, Bruno, Claudio, Corti, Stefania, Masson, Riccardo, Straub, Volker, Vita, Giuseppe, Ernst, Uwe, Kernbauer, Elaine, Williamson, Susan, Ouyang, Haojun, Feltner, Douglas E., Meriggioli, Matthew, Lavrov, Arseniy, L'Italien, James, Sproule, Douglas M., and Mercuri, Eugenio

Subjects
SPINAL muscular atrophy, MOTOR neurons
Published
2019
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14. A Phase 2, Placebo-Controlled Study of the Opioid Receptor Antagonist LY2196044 for the Treatment of Alcohol Dependence

Author

Wong, Conrad J., primary, Witcher, Jennifer, additional, Mallinckrodt, Craig, additional, Dean, Robert A., additional, Anton, Raymond F., additional, Chen, Yunfei, additional, Fijal, Bonnie A., additional, Ouyang, Haojun, additional, Dharia, Sweta, additional, Sundseth, Scott S., additional, Schuh, Kory J., additional, and Kinon, Bruce J., additional

Published
2013
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16. 250 AVXS-101 phase 3 study in spinal muscular atrophy type 1

Author

Day, John W, Chiriboga, Claudia A, Crawford, Thomas O, Darras, Basil T, Finkel, Richard S, Connolly, Anne M, Iannaccone, Susan T, Kuntz, Nancy L, Peña, Loren DM, Schultz, Meredith, Shieh, Perry B, Smith, Edward C, Kausar, Imran, Feltner, Douglas E, Ogrinc, Francis G, Ouyang, Haojun, Macek, Thomas A, Kernbauer, Elaine, Muehring, Lynlee M, L’Italien, James, Sproule, Douglas M, Kaspar, Brian K, and Mendell, Jerry R

Abstract

BackgroundOnasemnogene abeparvovec (AVXS-101), a one-time intravenous gene-replacement therapy, treats the genetic root cause of spinal muscular atrophy (SMA). In the phase 1 study, AVXS-101 dramatically improved survival, motor function, and motor milestone achievement in SMA type 1 (SMA1) patients.MethodsSTR1VE is a phase 3, multicenter, open-label study (NCT03306277) in SMA1 patients <6 months (bi-allelic survival motor neuron 1 gene [SMN1] mutations/deletions, 2 SMN2copies). Primary outcomes are independent sitting ≥30 seconds at 18 months of age, and survival (no death/permanent ventilation) at 14 months. Secondary outcomes include ability to thrive and ventilatory support at 18 months. Exploratory outcomes include CHOP-INTEND and Bayley-III.ResultsEnrollment is complete (22 patients dosed). Mean age at symptom onset, genetic diagnosis, and enrollment was 1.9 (0–4.0), 2.1 (0.5–4.0), and 3.7 (0.5–5.9) months. At baseline, no patient required ventilatory/nutritional support. Mean baseline CHOP-INTEND: 32.6 (17.0–52.0) points; increased by 6.9 (-4.0–16.0), 9.2 (0–18.0), and 11.7 (-3.0–23.0) points at 1, 2, and 3 months; independent sitting: 11/22 patients; survival at 13.6 months: 13/15 (87%) (8 March 2019 datacut).ConclusionsPreliminary data from the AVXS-101 phase 3 study show rapid motor function improvements in SMA1 patients, paralleling phase 1 findings.

Published
2019
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17. Bayesian Approach for Nonlinear Dynamic System and Genome-Wide Association Study

Author

Ouyang, Haojun

Subjects
dynamic system, multiple testing, genome-wide association study, heterogeneity, EM algorithm, meta-analysis, false discovery rate
Abstract

Genome-wide association studies (GWAS) have been widely used to identify single-nucleotide polymorphisms (SNPs) that are responsible for diseases. A challenging aspect of this study is to resolve the various issues related to multiple tests. We propose a new Bayesian method to measure statistical significance in these genome-wide studies based on the concept of false discovery rate (FDR). Our proposed method provides a convenient way to integrate prior knowledge obtained from external resources into current study. By controlling Bayesian positive FDR at a given level, the realized FDR is controlled. Our simulations show that the power can be substantially improved with correct prior information while the FDR is controlled at the desired level. When prior information is imprecise, our method can still improve the power of detecting signals, while keeping the FDR under control. The modified Bayesian method is applied to a GWAS for schizophrenia. Meta-analysis is another approach to utilize information from multiple sources by combining results from multiple independent studies. A major concern in carrying out meta-analysis involves the proper characterization of heterogeneity among population. To account for heterogeneity, the most commonly used approach is to implement a random-effects model, where the random-effects are assumed to be normally distributed with an unknown population mean and an unknown variance. We relax the normality assumption and show that a broad class of distributions can be approximated by a class of mixture distributions. The population mean and variance estimates based on the mixture density are then obtained by the EM algorithm. Our results show that the proposed method greatly improves the accuracy in estimating overall mean effect and heterogeneity variance in various realistic cases. We illustrate our method to a study on DRD2 gene in multiple association studies with schizophrenia. Dynamic system defined by ordinary differential equations is an important tool to modeling complicated biology system. To estimate parameters in the dynamic system which analytic, close form solution is not available and involving missing or censored data, we extend Bayesian Euler's Approximation method based on data augmentation algorithm. Our simulation study shown the method is robust in both cases. The proposed method is applied to analyze HIV viral load dataset, which enable us to retrieve information from the censored data.

Published
2010

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