PubMed Clinical Queries
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Results for Clinical Studies: Therapy/Broad
5 of 2,591 results sorted by: Most Recent
See all results in PubMed (2,591)-
Beneficial immune-modulatory effects of the N-163 strain of Aureobasidium pullulans-produced 1,3-1,6 Beta glucans in Duchenne muscular dystrophy: Results of an open-label, prospective, exploratory case-control clinical study.
Raghavan K, et al. IBRO Neurosci Rep. 2023. PMID: 38053632 Free PMC article.
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Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.
Shoti J, et al. Mol Ther Methods Clin Dev. 2023. PMID: 38046199 Free PMC article.
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Adeno-associated virus-mediated gene therapy for rare pediatric neurogenetic diseases: Current status and outlook.
Wang X, et al. Zhong Nan Da Xue Xue Bao Yi Xue Ban. 2023. PMID: 38044650 Free article. Chinese, English.
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Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy.
Engelbeen S, et al. Nucleic Acid Ther. 2023. PMID: 38010230 Free PMC article.
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Quantifying Variability in Motor Function in Duchenne Muscular Dystrophy: UK Centiles for the NorthStar Ambulatory Assessment, 10 m Walk Run Velocity and Rise from Floor Velocity in GC Treated Boys.
Stimpson G, et al. J Neuromuscul Dis. 2023. PMID: 37980680