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Journal of Neurology|May 26, 2026
Identification of prognostic biomarkers in a large cohort of patients with LGMD R2Carla F Bolano-Diaz, Jose Verdu-Diaz, Dan Hao, et al.Annals of Neurology|June 3, 2010
Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophyVinod Malik, Louise R Rodino-Klapac, Laurence Viollet, et al.Frontiers in Cell and Developmental Biology|July 27, 2023
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophyJerry R Mendell, Perry B Shieh, Craig M McDonald, et al.Research Square|July 28, 2023
Protein arginine methyltransferase 5 (PRMT5) is an actionable therapeutic target in CDK4/6 inhibitor-resistant ER+/RB-deficient breast cancerChang-Ching Lin, Tsung-Cheng Chang, Yunguan Wang, et al.Annals of Neurology|March 13, 2008
A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophyKathryn R Wagner, James L Fleckenstein, Anthony A Amato, et al.Nature Communications|March 14, 2024
PRMT5 is an actionable therapeutic target in CDK4/6 inhibitor-resistant ER+/RB-deficient breast cancerChang-Ching Lin, Tsung-Cheng Chang, Yunguan Wang, et al.Nature Communications|September 29, 2025
Post-resection delivery of a TLR7/8 agonist from a biodegradable scaffold achieves immune-mediated glioblastoma clearance and protection against tumor challenge in miceElizabeth G Graham-Gurysh, Ryan N Woodring, Sean R Simpson, et al.Neuromuscular Disorders : NMD|March 27, 2023
Expanding the muscle imaging spectrum in dysferlinopathy: description of an outlier population from the classical MRI patternLaura Llansó, Ursula Moore, Carla Bolano-Diaz, et al.The Lancet. Neurology|March 20, 2021
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trialJohn W Day, Richard S Finkel, Claudia A Chiriboga, et al.Nature Medicine|October 9, 2024
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trialJerry R Mendell, Francesco Muntoni, Craig M McDonald, et al.Pageof 89