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Journal of Neuromuscular Diseases|December 3, 2016
European Cross-Sectional Survey of Current Care Practices for Duchenne Muscular Dystrophy Reveals Regional and Age-Dependent DifferencesJulia Vry, Kathrin Gramsch, Sunil Rodger, et al.
Neuromuscular Disorders : NMD|January 2, 2018
Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional careEugenio Mercuri, Richard S Finkel, Francesco Muntoni, et al.
Neuromuscular Disorders : NMD|January 7, 2018
Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethicsRichard S Finkel, Eugenio Mercuri, Oscar H Meyer, et al.
Cell Stem Cell|May 14, 2026
Genotoxicity profiling reveals distinct platform-and cell type-specific effects in therapeutic gene editing for genetic hyperinflammationLei Lei, Masako M Kaufmann, Jessica Lao, et al.
The New England Journal of Medicine|February 15, 2018
Nusinersen versus Sham Control in Later-Onset Spinal Muscular AtrophyEugenio Mercuri, Basil T Darras, Claudia A Chiriboga, et al.
Human Mutation|June 3, 2018
Targeted sequencing with expanded gene profile enables high diagnostic yield in non-5q-spinal muscular atrophiesMert Karakaya, Markus Storbeck, Eike A Strathmann, et al.
Cell Reports. Medicine|June 19, 2024
Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseasesFederica Esposito, Fabio Dell'Aquila, Manuel Rhiel, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 16, 2025
Targeting BCL11B in CAR-engineered lymphoid progenitors drives NK-like cell development with prolonged anti-leukemic activityFranziska Baatz, Arnab Ghosh, Jessica Herbst, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 1, 2022
Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosaJohannes Bischof, Oliver Patrick March, Bernadette Liemberger, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 1, 2025
Prime editing as a promising therapeutic strategy for junctional epidermolysis bullosaMarkus Hierl, Johannes Bischof, Bernadette Liemberger, et al.
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