Author

X. Nissan

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Review Open access Sep 2026

Bridging the translational gap in drug repurposing for rare diseases: clinical success versus regulatory conversion.

Repurposing of drugs to treat rare diseases (RDs) is vital, but the translational gap between clinical evidence and regulatory approval remains unquantified. Analysis of 47 clinical trials showed that 66 % of completed studies met their primary or key secondary endpoints. Nevertheless, only 39 % achieved marketing auth...

Wael Khazen, Arnaud Valent, Samantha Parker et al. · Drug Discovery Today · 0 citations

Open access Aug 2026

Generation and characterization of three human induced pluripotent stem cell lines from patients with Duchenne muscular dystrophy.

Three induced pluripotent stem cell lines reprogrammed from peripheral blood mononuclear cells (PBMCs) of patients with DMD constitute a relevant human cellular model for studying disease-associated phenotypes and evaluating potential therapeutic strategies.

Hamel Mahiou, Benjamin Marande, Lina El Kassar et al. · Stem Cell Research · 0 citations