Neuropediatrics

Issue S 02 · Volume 50 · September 2019 DOI: 10.1055/s-009-44190

Abstracts of the 45th Annual Meeting of the Society for Neuropediatrics
München, 11.09 – 14.09.2019

Chair: Prof. Dr. Martin Staudt, Pediatric Neurology, Neurorehabilitation and Epileptology, Schön Klinik Vogtareuth

Abstracts (HTML) List of Authors

Oral Presentations

Neuromuscular Diseases and Varia

GNP-FV14

Schultz, Meredith; Swoboda, Kathryn J.; Farrar, Michelle; McMillan, Hugh; Parsons, Julie; Ernst, Uwe; Kernbauer, Elaine; Farrow, Marcia; Ogrinc, Francis G.; Kavanagh, Sarah; Feltner, Douglas E.; McGill, Bryan E.; Spector, Sidney A.; L’Italien, James; Sproule, Douglas; Strauss, Kevin:

Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) in Pre-symptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update

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GNP-FV15

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; Schultz, Meredith; Shieh, Perry B.; Smith, Edward C.; Ernst, Uwe; Feltner, Douglas E.; Orgrinc, Francis G.; Shah, Ankita; Ouyang, Haojun; Macek, Thomas A.; Kernbauer, Elaine; L’Italien, James; Sproule, Douglas M.; Kaspar, Brian K.; Medel, Jerry R.:

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

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GNP-FV16

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.; Mercuri, Eugenio:

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

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GNP-FV17

Müller-York, André; Iff, Joel; Gerrits, Charles; Sajeev, Gautam; Signorovitch, James; Tuttle, Edward; Birk, Erica; Hossain, Intekhab; Yao, Zhiwen; Ng, Courtney; Goemans, Nathalie:

Eteplirsen Delays Time to Loss of Ambulation in Patients With Duchenne Muscular Dystrophy Compared With Patients Receiving Standard of Care

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GNP-FV18

Regensburger, Adrian P.; Fonteyne, Lina; Jüngert, Jörg; Qurashi, Matthias; Neurath, Markus F.; Klymiuk, Nikolai; Kemter, Elisabeth; Rascher, Wolfgang; Trollmann, Regina; Wolf, Eckhard; Waldner, Maximilian J.; Knieling, Ferdinand: