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Filter Applied: gene therapy (Click to remove)

Current and Emerging Issues in Wilsons Disease
NEJM 389:922-938, Roberts,E.A. & Schilsky, M.L., 2023

Muscular Dystrophies
Lancet 394:2025-2038, Mercuri, E.,et al, 2019

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
NEJM 377:1713-1722,1786, Mendell, J.R.,et al, 2017

Diagnosis and New Treatments in Muscular Dystrophies
JNNP 80:706-714, Manzur,A.Y. &Muntoni,F., 2009

Glycogen-Storage Disease Type II
eMedicine, May 2, Ibrahim,J. &McGovern,M., 2006

Long-Term Safety and Tolerability of ProSavin, a Lentiviral Vector-Based Gene Therapy for Parkinsons Disease: A Dose Escalation, Open-Label, Phase 1/2 Trial
Lancet 383:1138-1146,1107, Palfi, S.,et al, 2014

Spinal Muscular Atrophy A Timely Review
Arch Neurol 68:979-984, Kolb, S.J.,et al, 2011



Showing articles 0 to 7 of 7