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Author/Creator Burghes, Arthur H.M.

1. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones. (June 2017)

Authors:
Mendell, Jerry R.; Al-Zaidy, Samiah; Shell, Richard; Arnold, W. David; Rodino-Klapac, Louise; Prior, Thomas W.; Lowes, Linda; Alfano, Lindsay; Berry, Katherine; Church, Kathleen; Kissel, John T.; Nagendran, Sukumar; L'Italien, James; Sproule, Douglas M.; Cardenas, Jessica; Burghes, Arthur H.M.; F...
Journal:
European journal of paediatric neurology
Issue:
Volume 21(2017)Supplement 1
Page Start:
e13
Record Type:
Journal Article
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Available online (eLD content is only available in our Reading Rooms) ↗

2. Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Issue 2 (21st January 2016)

Authors:
Kolb, Stephen J.; Coffey, Christopher S.; Yankey, Jon W.; Krosschell, Kristin; Arnold, W. David; Rutkove, Seward B.; Swoboda, Kathryn J.; Reyna, Sandra P.; Sakonju, Ai; Darras, Basil T.; Shell, Richard; Kuntz, Nancy; Castro, Diana; Iannaccone, Susan T.; Parsons, Julie; Connolly, Anne M.; Chiribog...
Journal:
Annals of clinical and translational neurology
Issue:
Volume 3:Issue 2(2016)
Page Start:
132
Record Type:
Journal Article
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Available online (eLD content is only available in our Reading Rooms) ↗

3. Natural history of infantile‐onset spinal muscular atrophy. Issue 6 (8th December 2017)

Authors:
Kolb, Stephen J.; Coffey, Christopher S.; Yankey, Jon W.; Krosschell, Kristin; Arnold, W. David; Rutkove, Seward B.; Swoboda, Kathryn J.; Reyna, Sandra P.; Sakonju, Ai; Darras, Basil T.; Shell, Richard; Kuntz, Nancy; Castro, Diana; Parsons, Julie; Connolly, Anne M.; Chiriboga, Claudia A.; McDonal...
Journal:
Annals of neurology
Issue:
Volume 82:Issue 6(2017)
Page Start:
883
Record Type:
Journal Article
View Content:
Available online (eLD content is only available in our Reading Rooms) ↗