P915: Longitudinal motor unit number estimation in a spinal muscular atrophy cohort
Kaufmann, P, Kang, P.B, Gooch, C.L, McDermott, M.P, Darras, B.T, Finkel, R.S, Yang, M.L, Sproule, D.M, Chung, W.K, De Vivo, D.C, M.S.G. Pediatric Neuromuscular Clinical Research Network
Published in Clinical neurophysiology (01.06.2014)
Published in Clinical neurophysiology (01.06.2014)
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PND24 Burden of Illness of Spinal Muscular Atrophy: An Update
Droege, M., Dabbous, O., Arjunji, R., Patel, A., Gauthier-Loiselle, M., Cloutier, M., Sproule, D.M.
Published in Value in health (01.12.2020)
Published in Value in health (01.12.2020)
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Burden of illness of spinal muscular atrophy type 1 (SMA1)
Droege, M., Dabbous, O., Arjunji, R., Seda, J., Gauthier-Loiselle, M., Cloutier, M., Sproule, D.M.
Published in Journal of the neurological sciences (15.10.2019)
Published in Journal of the neurological sciences (15.10.2019)
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PRO51 COST-EFFECTIVENESS AND BUDGET IMPACT OF ONASEMNOGENE ABEPARVOVEC FOR SPINAL MUSCULAR ATROPHY TYPE 1: POST-HOC ANALYSIS OF A MODEL DEVELOPED BY ICER
Dabbous, O., Dean, R., Arjunji, R., Sproule, D.M., Feltner, D.E., Malone, D.C.
Published in Value in health (01.11.2019)
Published in Value in health (01.11.2019)
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PRO22 BURDEN OF ILLNESS OF SPINAL MUSCULAR ATROPHY: AN UPDATE
Droege, M., Dabbous, O., Arjunji, R., Gauthier-Loiselle, M., Cloutier, M., Sproule, D.M.
Published in Value in health (01.05.2019)
Published in Value in health (01.05.2019)
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PMU36 COST-UTILITY ANALYSIS OF SINGLE DOSE GENE-REPLACEMENT THERAPY FOR SPINAL MUSCULAR ATROPHY TYPE 1 COMPARED TO CHRONIC NUSINERSEN TREATMENT IN JAPAN
Dean, R., Miller, B., Arjunji, R., Awano, H., Igarashi, A., Tanaka, S., Feltner, D.E., Sproule, D.M., Jensen, I., Dabbous, O.
Published in Value in health (01.05.2020)
Published in Value in health (01.05.2020)
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PSY18 - RAPID IMPROVEMENTS IN MOTOR FUNCTION IN SPINAL MUSCULAR ATROPHY TYPE 1 FOLLOWING AVXS-101 GENE REPLACEMENT THERAPY
Dabbous, O., Droege, M., Feltner, D., Novack, A., Menier, M., Ryman, D., Sproule, D.M.
Published in Value in health (01.10.2018)
Published in Value in health (01.10.2018)
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PND11 - INDIRECT TREATMENT COMPARISON OF AVXS-101 TO NUSINERSEN FOR THE TREATMENT OF TYPE 1 SPINAL MUSCULAR ATROPHY
Lorenzi, M, Dabbous, O, Jansen, JP, Maru, B, Sproule, D.M., Feltner, D, White, C
Published in Value in health (01.10.2018)
Published in Value in health (01.10.2018)
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Natural history of MELAS associated with mitochondrial DNA m.3243A>G genotype
Kaufmann, P, Engelstad, K, Wei, Y, Kulikova, R, Oskoui, M, Sproule, D M, Battista, V, Koenigsberger, D Y, Pascual, J M, Shanske, S, Sano, M, Mao, X, Hirano, M, Shungu, D C, Dimauro, S, De Vivo, D C
Published in Neurology (29.11.2011)
Published in Neurology (29.11.2011)
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PRO2 THE VALUE OF ONASEMNOGENE ABEPARVOVEC GENE-REPLACEMENT THERAPY IN IMPROVING SURVIVAL AND MOTOR FUNCTION AND DECREASING VENTILATORY SUPPORT AND HOSPITALIZATION CONTRASTED TO NATURAL HISTORY FOR SPINAL MUSCULAR ATROPHY TYPE 1
Dabbous, O., Sproule, D.M., Feltner, D.E., Ogrinc, F.G., Menier, M., Droege, M., Maru, B., Khan, F., Arjunji, R.
Published in Value in health (01.11.2019)
Published in Value in health (01.11.2019)
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Journal Article
PBI10 TYPE I SPINAL MUSCULAR ATROPHY PATIENTS TREATED WITH AVXS-101 HAVE LOWER USE OF VENTILATORY SUPPORT, HOSPITALIZATION, AND ASSOCIATED COSTS COMPARED TO THOSE TREATED WITH NUSINERSEN
Arjunji, R., Dean, R., Jensen, I.S., Miller, B., Menier, M., Sproule, D.M., Feltner, D.E., Droege, M., Khan, F., Dabbous, O.
Published in Value in health (01.05.2019)
Published in Value in health (01.05.2019)
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PND9 EVENT-FREE SURVIVAL AND MOTOR MILESTONE ACHIEVEMENT FOLLOWING AVXS-101 AND NUSINERSEN INTERVENTIONS CONTRASTED TO NATURAL HISTORY FOR TYPE I SPINAL MUSCULAR ATROPHY PATIENTS
Dabbous, O., Sproule, D.M., Feltner, D.E., Ogrinc, F.G., Menier, M., Droege, M., Maru, B., Khan, F., Arjunji, R.
Published in Value in health (01.05.2019)
Published in Value in health (01.05.2019)
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ND2 COST-UTILITY ANALYSIS OF SINGLE DOSE GENE-REPLACEMENT THERAPY FOR SPINAL MUSCULAR ATROPHY TYPE 1 COMPARED TO CHRONIC NUSINERSEN TREATMENT
Malone, D., Dean, R., Miller, B., Arjunji, R., Feltner, D.E., Sproule, D.M., Jensen, I.S., Maru, B., Dabbous, O.
Published in Value in health (01.05.2019)
Published in Value in health (01.05.2019)
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Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial
Mendell, J.R., Shell, R., Lehman, K.J., McColly, M., Lowes, L.P., Alfano, L.N., Miller, N.F., Iammarino, M.A., Church, K., Spector, S.A., Ogrinc, F.G., Ouyang, H., Kernbauer, E., Shah, S., L’Italien, J., Sproule, D.M., Feltner, D.E., Al-Zaidy, S.
Published in Journal of the neurological sciences (15.10.2019)
Published in Journal of the neurological sciences (15.10.2019)
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PND13 Burden of Illness of Spinal Muscular Atrophy: An Update
Droege, M., Dabbous, O., Arjunji, R., Gauthier-Loiselle, M., Cloutier, M., Sproule, D.M.
Published in Value in health regional issues (01.09.2020)
Published in Value in health regional issues (01.09.2020)
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Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): Global pivotal phase 3 study program (STR1VE-US, STR1VE-EU, STR1VE-AP)
Mercuri, E., Baranello, G., Day, J.W., Bruno, C., Corti, S., Chiriboga, C.A., Crawford, T.O., Darras, B.T., Finkel, R.S., Connolly, A.M., Iannaccone, S.T., Kuntz, N.L., Masson, R., Peña, L.D.M., Baldinetti, F., Schultz, M., Shieh, P.B., Smith, E.C., Saito, K., Scoto, M., Spector, S.A., Truncated, A. Authors, Sproule, D.M., Mendell, J.R., Muntoni, F.
Published in Journal of the neurological sciences (15.10.2019)
Published in Journal of the neurological sciences (15.10.2019)
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Intrathecal administration of AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): Phase 1/2A study (strong)
Finkel, R.S., Day, J.W., Darras, B.T., Kuntz, N.L., Connolly, A.M., Crawford, T.O., Butterfield, R.J., Shieh, P.B., Tennekoon, G., Iannaccone, S.T., Meriggioli, M., Ogrinc, F.G., Kavanagh, S., Kernbauer, E., Whittle, J., L'Italien, J., Kaspar, B., Sproule, D.M., Spector, S.A., Feltner, D.E., Mendell, J.R.
Published in Journal of the neurological sciences (15.10.2019)
Published in Journal of the neurological sciences (15.10.2019)
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Onasemnogene abeparvovec gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update
Strauss, K.A., Swoboda, K.J., Farrar, M.A., McMillan, H.J., Parsons, J., Krueger, J.M., Iannaccone, S.T., Chiriboga, C.A., Kwon, J.M., Saito, K., Scoto, M., Baldinetti, F., Schultz, M., Kernbauer, E., Farrow, M., Ogrinc, F.G., Kavanagh, S., Feltner, D.E., McGill, B.E., Spector, S.A., L’Italien, J., Sproule, D.M., Muntoni, F.
Published in Journal of the neurological sciences (15.10.2019)
Published in Journal of the neurological sciences (15.10.2019)
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PRO6 BURDEN OF ILLNESS OF SPINAL MUSCULAR ATROPHY TYPE 1
Droege, M., Dabbous, O., Arjunji, R., Seda, J., Gauthier-Loiselle, M., Cloutier, M., Sproule, D.M.
Published in Value in health regional issues (01.10.2019)
Published in Value in health regional issues (01.10.2019)
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