A newly approved high-dose Spinraza regimen may offer added SMA treatment benefits, with similar safety findings to the ...

In vitro data demonstratedself-complementary AAV9 (scAAV9) enabled ~30-fold higher MeCP2 protein expression compared to single-stranded AAV9 ...

Molecular-cloning studies have revealed the existence of five molecularly distinct mammalian muscarinic acetylcholine receptors (mAChRs), M 1 –M 5, which are all members of the superfamily of class I ...

Lipid nanoparticles address key drug delivery challenges, including immune evasion, toxicity, and regulatory approval.