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1

دورية أكاديمية

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

المؤلفون: Masson R., Mazurkiewicz-Beldzinska M., Rose K., Servais L., Xiong H., Zanoteli E., Baranello G., Bruno C., Day J. W., Deconinck N., Klein A., Mercuri E., Vlodavets D., Wang Y., Dodman A., El-Khairi M., Gorni K., Jaber B., Kletzl H., Gaki E., Fontoura P., Darras B. T., Volpe J. J., Posner J., Kellner U., Quinlivan R., Gerber M., Khwaja O., Scalco R. S., Seabrook T., Koch A., Balikova I., Joniau I., Accou G., Tahon V., Wittevrongel S., De Vos E., de Holanda Mendonca R., Matsui Jr C., Fornazieri Darcie A. L., Machado C., Kiyoko Oyamada M., Martini J., Polido G., Rodrigues Iannicelli J., Caires de Oliveira Achili Ferreira J., Hu C., Zhu X., Qian C., Shen L., Li H., Shi Y., Zhou S., Xiao Y., Zhou Z., Wang S., Sang T., Wei C., Dong H., Cao Y., Wen J., Li W., Qin L., Barisic N., Celovec I., Galiot Delic M., Ivkic P. K., Vukojevic N., Kern I., Najdanovic B., Skugor M., Tomas J., Boespflug-Tanguy O., De Lucia S., Seferian A., Barreau E., Mnafek N., Peche H., Grange A., Trang Nguyen D., Milascevic D., Tachibana S., Pagliano E., Bianchi Marzoli S., Santarsiero D., Garcia Sierra M., Tremolada G., Arnoldi M. T., Vigano M., Dosi C., Zanin R., Schembri V., Brolatti N., Rao G., Tassara E., Morando S., Tacchetti P., Pedemonte M., Priolo E., Sposetti L., Comi G. P., Govoni A., Osnaghi S. G., Minorini V., Abbati F., Fassini F., Foa M., Lopopolo A., Pane M., Palermo C., Pera M. C., Amorelli G. M., Barresi C., D'Amico G., Orazi L., Coratti G., Leone D., Laura A., De Sanctis R., Berti B., Kimura N., Takeshima Y., Shimomura H., Lee T., Gomi F., Morimatsu T., Furukawa T., Stodolska-Koberda U., Waskowska A., Kolendo J., Sobierajska-Rek A., Modrzejewska S., Lemska A., Melnik E., Artemyeva S., Leppenen N., Yupatova N., Monakhova A., Papina Y., Shidlovsckaia O., Litvinova E., Enzmann C., Galiart E., Gugleta K., Wondrusch Haschke C., Topaloglu H., Oncel I., Ertugrul N. E., Konuskan B., Eldem B., Kadayifcilar S., Alemdaroglu I., Sari S., Bilgin N., Karaduman A. A., Sarikaya F. G. Y., Graham R. J., Ghosh P., Casavant D., Levine A., Titus R., Engelbrekt A., Ambrosio L., Fulton A., Baglieri A. M., Dias C., Maczek E., Pasternak A., Beres S., Duong T., Gee R., Young S.

المساهمون: Masson, R., Mazurkiewicz-Beldzinska, M., Rose, K., Servais, L., Xiong, H., Zanoteli, E., Baranello, G., Bruno, C., Day, J. W., Deconinck, N., Klein, A., Mercuri, E., Vlodavets, D., Wang, Y., Dodman, A., El-Khairi, M., Gorni, K., Jaber, B., Kletzl, H., Gaki, E., Fontoura, P., Darras, B. T., Volpe, J. J., Posner, J., Kellner, U., Quinlivan, R., Gerber, M., Khwaja, O., Scalco, R. S., Seabrook, T., Koch, A., Balikova, I., Joniau, I., Accou, G., Tahon, V., Wittevrongel, S., De Vos, E., de Holanda Mendonca, R., Matsui Jr, C., Fornazieri Darcie, A. L., Machado, C., Kiyoko Oyamada, M., Martini, J., Polido, G., Rodrigues Iannicelli, J., Caires de Oliveira Achili Ferreira, J., Hu, C., Zhu, X., Qian, C., Shen, L., Li, H., Shi, Y., Zhou, S., Xiao, Y., Zhou, Z., Wang, S., Sang, T., Wei, C., Dong, H., Cao, Y., Wen, J., Li, W., Qin, L., Barisic, N., Celovec, I., Galiot Delic, M., Ivkic, P. K., Vukojevic, N., Kern, I., Najdanovic, B., Skugor, M., Tomas, J., Boespflug-Tanguy, O., De Lucia, S., Seferian, A., Barreau, E., Mnafek, N., Peche, H., Grange, A., Trang Nguyen, D., Milascevic, D., Tachibana, S., Pagliano, E., Bianchi Marzoli, S., Santarsiero, D., Garcia Sierra, M., Tremolada, G., Arnoldi, M. T., Vigano, M., Dosi, C., Zanin, R., Schembri, V., Brolatti, N., Rao, G., Tassara, E., Morando, S., Tacchetti, P., Pedemonte, M., Priolo, E., Sposetti, L.

وصف الملف: ELETTRONICO

العلاقة: info:eu-repo/semantics/altIdentifier/wos/WOS:000898849800021; volume:21; firstpage:1110; lastpage:1119; numberofpages:10; journal:LANCET NEUROLOGY; https://hdl.handle.net/11567/1143175Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85141001742

الإتاحة: https://doi.org/10.1016/S1474-4422Test(22)00339-8
https://hdl.handle.net/11567/1143175Test

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2

دورية أكاديمية

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

المؤلفون: Masson, R, Mazurkiewicz-Bełdzińska, M, Rose, K, Servais, L, Xiong, H, Zanoteli, E, Baranello, G, Bruno, C, Day, JW, Deconinck, N, Klein, A, Mercuri, E, Vlodavets, D, Wang, Y, Dodman, A, El-Khairi, M, Gorni, K, Jaber, B, Kletzl, H, Gaki, E, Fontoura, P, Darras, BT, Volpe, JJ, Posner, J, Kellner, U, Quinlivan, R, Gerber, M, Khwaja, O, Scalco, RS, Seabrook, T, Koch, A, Balikova, I, Joniau, I, Accou, G, Tahon, V, Wittevrongel, S, De Vos, E, de Holanda Mendonça, R, Matsui, C, Fornazieri Darcie, AL, Machado, C, Kiyoko Oyamada, M, Martini, J, Polido, G, Rodrigues Iannicelli, J, Caires de Oliveira Achili Ferreira, J, Hu, C, Zhu, X, Qian, C, Shen, L, Li, H, Shi, Y, Zhou, S, Xiao, Y, Zhou, Z, Wang, S, Sang, T, Wei, C, Dong, H, Cao, Y, Wen, J, Li, W, Qin, L, Barisic, N, Celovec, I, Galiot Delic, M, Ivkic, PK, Vukojevic, N, Kern, I, Najdanovic, B, Skugor, M, Tomas, J, Boespflug-Tanguy, O, De Lucia, S, Seferian, A, Barreau, E, Mnafek, N, Peche, H, Grange, A, Trang Nguyen, D, Milascevic, D, Tachibana, S, Pagliano, E, Bianchi Marzoli, S, Santarsiero, D, Garcia Sierra, M, Tremolada, G, Arnoldi, MT, Vigano, M, Dosi, C, Zanin, R, Schembri, V, Brolatti, N, Rao, G, Tassara, E, Morando, S, Tacchetti, P, Pedemonte, M, Priolo, E, Sposetti, L

المصدر: The Lancet Neurology , 21 (12) pp. 1110-1119. (2022)

مصطلحات موضوعية: FIREFISH Study Group

وصف الملف: text

العلاقة: https://discovery.ucl.ac.uk/id/eprint/10160515/2/Baranello_THELANCETNEUROLOGY-D-22-00295R1_manuscript_tracked_20220808_to%20page%20proofs.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10160515Test/

الإتاحة: https://discovery.ucl.ac.uk/id/eprint/10160515/2/Baranello_THELANCETNEUROLOGY-D-22-00295R1_manuscript_tracked_20220808_to%20page%20proofs.pdfTest
https://discovery.ucl.ac.uk/id/eprint/10160515Test/

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3

دورية أكاديمية

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

المؤلفون: Darras, BT, Masson, R, Mazurkiewicz-Bełdzińska, M, Rose, K, Xiong, H, Zanoteli, E, Baranello, G, Bruno, C, Vlodavets, D, Wang, Y, El-Khairi, M, Gerber, M, Gorni, K, Khwaja, O, Kletzl, H, Scalco, RS, Fontoura, P, Servais, L, FIREFISH Working Group

المصدر: New England Journal of Medicine , 385 pp. 427-435. (2021)

وصف الملف: text

العلاقة: https://discovery.ucl.ac.uk/id/eprint/10132424/1/nejmoa2102047.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10132424Test/

الإتاحة: https://discovery.ucl.ac.uk/id/eprint/10132424/1/nejmoa2102047.pdfTest
https://discovery.ucl.ac.uk/id/eprint/10132424Test/

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4

دورية أكاديمية

Risdiplam in Type 1 Spinal Muscular Atrophy

المؤلفون: Baranello, G, Darras, BT, Day, JW, Deconinck, N, Klein, A, Masson, R, Mercuri, E, Rose, K, El-Khairi, M, Gerber, M, Gorni, K, Khwaja, O, Kletzl, H, Scalco, RS, Seabrook, T, Fontoura, P, Servais, L, FIREFISH Working Group

المصدر: New England Journal of Medicine , 384 (10) pp. 915-923. (2021)

مصطلحات موضوعية: Administration, Oral, Azo Compounds, Dose-Response Relationship, Drug, Female, Humans, Infant, Male, Neuromuscular Agents, Progression-Free Survival, Pyrimidines, RNA Splicing, Respiratory Insufficiency, Respiratory Tract Infections, Spinal Muscular Atrophies of Childhood, Survival of Motor Neuron 1 Protein

وصف الملف: text

العلاقة: https://discovery.ucl.ac.uk/id/eprint/10129084/1/Baranello_nejmoa2009965.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10129084Test/

الإتاحة: https://discovery.ucl.ac.uk/id/eprint/10129084/1/Baranello_nejmoa2009965.pdfTest
https://discovery.ucl.ac.uk/id/eprint/10129084Test/

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5

دورية أكاديمية

CLINICAL TRIAL HIGHLIGHTS

المؤلفون: Servais, L., Al-Muhaizea, M., Farrar, M., Nelson, L., Prufer, A., Finkel, R., Wang, Y., Zanoteli, E., El-Khairi, M., Gerber, M., Gorni, K., Kletzl, H., Palfreeman, L., Scalco, R., Bertini, E.

المصدر: Neuromuscular Disorders ; volume 31, page S48 ; ISSN 0960-8966

مصطلحات موضوعية: Genetics (clinical), Neurology (clinical), Neurology, Pediatrics, Perinatology and Child Health

الإتاحة: https://doi.org/10.1016/j.nmd.2021.07.021Test
https://api.elsevier.com/content/article/PII:S096089662100208X?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S096089662100208X?httpAccept=text/plainTest

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6

دورية أكاديمية

SMA - TREATMENT

المؤلفون: Masson, R., Boespflug-Tanguy, O., Darras, B., Day, J., Deconinck, N., Klein, A., Mazurkiewicz-Bełdzińska, M., Mercuri, E., Rose, K., Servais, L., Vlodavets, D., Xiong, H., Zanoteli, E., Dodman, A., El-Khairi, M., Gaki, E., Gerber, M., Gorni, K., Kletzl, H., Baranello, G.

المصدر: Neuromuscular Disorders ; volume 31, page S134 ; ISSN 0960-8966

مصطلحات موضوعية: Genetics (clinical), Neurology (clinical), Neurology, Pediatrics, Perinatology and Child Health

الإتاحة: https://doi.org/10.1016/j.nmd.2021.07.303Test
https://api.elsevier.com/content/article/PII:S0960896621004909?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0960896621004909?httpAccept=text/plainTest

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7

دورية أكاديمية

SMA - TREATMENT

المؤلفون: Chiriboga, C., Bruno, C., Duong, T., Fischer, D., Kirschner, J., Mercuri, E., Gerber, M., Gorni, K., Kletzl, H., Carruthers, I., Martin, C., Warren, F., Scoto, M.

المصدر: Neuromuscular Disorders ; volume 31, page S134-S135 ; ISSN 0960-8966

مصطلحات موضوعية: Genetics (clinical), Neurology (clinical), Neurology, Pediatrics, Perinatology and Child Health

الإتاحة: https://doi.org/10.1016/j.nmd.2021.07.304Test
https://api.elsevier.com/content/article/PII:S0960896621004910?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0960896621004910?httpAccept=text/plainTest

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8

دورية أكاديمية

PII-059* Mechanistic Population Pharmacokinetic Modeling of Risdiplam for Extrapolation of Exposure and Cyp3a Drug-drug Interaction in Neonates with Spinal Muscular Atrophy

المؤلفون: Cleary, Y, Kletzl, H, Grimsey, P, Heinig, K, Ogungbenro, K, Silber--Baumann, H, Frey, N, Aarons, L, Galetin, A, Gertz, M

المصدر: Cleary , Y , Kletzl , H , Grimsey , P , Heinig , K , Ogungbenro , K , Silber--Baumann , H , Frey , N , Aarons , L , Galetin , A & Gertz , M 2023 , ' PII-059* Mechanistic Population Pharmacokinetic Modeling of Risdiplam for Extrapolation of Exposure and Cyp3a Drug-drug Interaction in Neonates with Spinal Muscular Atrophy ' , Clinical Pharmacology & Therapeutics , vol. 113 , pp. S63 . https://doi.org/10.1002/cpt.2835Test

العلاقة: https://research.manchester.ac.uk/en/publications/1b092e57-a35c-4c6b-9b36-74fa4d4d926dTest

الإتاحة: https://doi.org/10.1002/cpt.2835Test
https://research.manchester.ac.uk/en/publications/1b092e57-a35c-4c6b-9b36-74fa4d4d926dTest
https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=pure_starter&SrcAuth=WosAPI&KeyUT=WOS:001002471100208&DestLinkType=FullRecord&DestApp=WOSTest

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