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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 NA...
by
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
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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
by
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
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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
by
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
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Onasemnogene abeparvovec gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update
by
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
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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)
by
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
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CM4 TIME TO TREATMENT EFFECT, EVENT-FREE SURVIVAL, AND MOTOR MILESTONE ACHIEVEMENT IN TYPE I SPINAL MUSCULAR ATROPHY PATIENTS TREATED WITH ONASEMNOGENE ABEPARVOVEC (AVXS-101) OR NU...
by
Dabbous, O.
,
Sproule, D.M.
,
Feltner, D.E.
,
Ogrinc, F.G.
,
Menier, M.
,
Droege, M.
,
Maru, B.
,
Khan, F.
,
Novack, A.
,
Arjunji, R.
Published in
Value in health regional issues
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