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UnknownFirst-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
Not available
S-EPMC9734078 | biostudies-literature
UnknownEculizumab improves fatigue in refractory generalized myasthenia gravis.
Not available
S-EPMC6620379 | biostudies-literature
UnknownGWAS of QRS duration identifies new loci specific to Hispanic/Latino populations.
Not available
S-EPMC6599128 | biostudies-literature
UnknownPlasma creatinine and oxidative stress biomarkers in amyotrophic lateral sclerosis.
Not available
S-EPMC7373369 | biostudies-literature
UnknownIdentification of asthma phenotypes using cluster analysis in the Severe Asthma Research Program.
Not available
S-EPMC2822971 | biostudies-literature
UnknownMachine learning suggests polygenic risk for cognitive dysfunction in amyotrophic lateral sclerosis.
Not available
S-EPMC7799365 | biostudies-literature
UnknownValidation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS.
Not available
S-EPMC7371380 | biostudies-literature
UnknownA Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS.
Not available
S-EPMC8117790 | biostudies-literature
UnknownGrowth in children with nephrotic syndrome: a post hoc analysis of the NEPTUNE study.
Not available
S-EPMC11728624 | biostudies-literature
UnknownConsistent improvement with eculizumab across muscle groups in myasthenia gravis.
Not available
S-EPMC7448154 | biostudies-literature
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