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DEVOTE 232SM203: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
Kuntz, N. L. (PD/PI)
3/27/20 → 8/31/50
Project: Research project
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An Open-Label, Expanded Access Protocol for Boys with Duchenne Muscular Dystrophy who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 Studies
Kuntz, N. L. (PD/PI)
1/8/20 → 8/31/50
Project: Research project
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A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101
Kuntz, N. L. (PD/PI)
10/29/19 → 8/31/50
Project: Research project
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SRK-015-002: PHASE 2 ACTIVE TREATMENT STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SRK-015 IN PATIENTS WITH LATER-ONSET SPINAL MUSCULAR ATROPHY (TOPAZ)
Kuntz, N. L. (PD/PI)
6/6/19 → 8/31/50
Project: Research project
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Sarepta 5051-102: An Open-Label Extension Study for Patients with Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
Kuntz, N. L. (PD/PI)
3/26/19 → 8/31/50
Project: Research project
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Solid Biosciences GX1001: A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and childrenwith Duchenne muscular dystrophy.
Kuntz, N. L. (PD/PI)
3/20/19 → 8/31/50
Project: Research project
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AveXis STRONG: A Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non-ambulatory Patients with Spinal Muscular Atrophy
Kuntz, N. L. (PD/PI)
10/4/17 → 8/31/50
Project: Research project
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A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients with Spinal Muscular Atrophy (SMA)
Kuntz, N. L. (PD/PI)
9/22/16 → 8/31/50
Project: Research project
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INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger
Kuntz, N. L. (PD/PI)
9/2/16 → 8/31/50
Project: Research project
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4045-101: A Randomized, Double Blind, Placebo Controlled, Dose Titration, Safety,Tolerability, and Pharmacokinetics Study Followed by an Open Label Safety and EfficacyEvaluation of SRP 4045 in Advanced Stage Patients with Duchenne Muscular Dyst
Kuntz, N. L. (PD/PI)
11/1/15 → 8/31/50
Project: Research project
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4045-101: A Randomized, Double Blind, Placebo Controlled, Dose Titration, Safety,Tolerability, and Pharmacokinetics Study Followed by an Open Label Safety and EfficacyEvaluation of SRP 4045 in Advanced Stage Patients with Duchenne Muscular Dyst
Kuntz, N. L. (PD/PI)
11/1/15 → 8/31/50
Project: Research project
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An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participatedin Investigational Studies of ISIS 396443
Kuntz, N. L. (PD/PI)
10/28/15 → 8/31/50
Project: Research project
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RECENSUS: A Medical Chart Review of Patients with X-Linked Myotubular Myopathy (XLMTM)
Kuntz, N. L. (PD/PI)
11/1/14 → 8/31/50
Project: Research project