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UnknownDetailed Biochemical and Bioenergetic Characterization of FBXL4-Related Encephalomyopathic Mitochondrial DNA Depletion.
Not available
S-EPMC5580732 | biostudies-literature
UnknownA Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial.
Not available
S-EPMC8543277 | biostudies-literature
UnknownA checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
Not available
S-EPMC5968578 | biostudies-literature
UnknownRecessive mutations in ATP8A2 cause severe hypotonia, cognitive impairment, hyperkinetic movement disorders and progressive optic atrophy.
Not available
S-EPMC6048855 | biostudies-literature
UnknownDeveloping standardized corticosteroid treatment for Duchenne muscular dystrophy.
Not available
S-EPMC6279424 | biostudies-literature
Unknown2'-OMe-phosphorodithioate-modified siRNAs show increased loading into the RISC complex and enhanced anti-tumour activity.
Not available
S-EPMC4112501 | biostudies-literature
UnknownNicotinic Receptors in the Brainstem Ascending Arousal System in SIDS With Analysis of Pre-natal Exposures to Maternal Smoking and Alcohol in High-Risk Populations of the Safe Passage Study.
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S-EPMC7988476 | biostudies-literature
UnknownMotor onset and diagnosis in Huntington disease using the diagnostic confidence level.
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S-EPMC4666501 | biostudies-literature
UnknownEfficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Not available
S-EPMC9425287 | biostudies-literature
UnknownTNG260 Is a Small-Molecule CoREST Inhibitor That Sensitizes STK11-Mutant Tumors to Anti-PD-1 Immunotherapy.
Not available
S-EPMC12521918 | biostudies-literature
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