2025
Bensimon G et Al. « "Efficacy and safety of low-dose IL-2 as an add-on therapy to riluzole (MIROCALS): a phase 2b, double-blind, randomised, placebo-controlled trial." » Lancet
Bruge C et Al.
« "High-Throughput Screening Identifies Bazedoxifene as a Potential Therapeutic for dysferlin-deficient limb girdle muscular dystrophy" »
Br J Pharmacol . 2025 Mar 19
Coppens S et Al.
« "Congenital Titinopathy: Comprehensive Characterization of the Most Severe End of the Disease Spectrum." »
Annals of Neurology 97(4): 611-628.
Di Leva F et Al.
« "SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems." »
Mol Ther 33(3): 1180-1196.
Hirt N et Al.
« "Systems immunology integrates the complex endotypes of recessive dystrophic epidermolysis bullosa." »
Nature communications 16(1): 664
