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

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

Multiple system atrophy (MSA) is an adult-onset, rapidly progressive neurodegenerative disorder characterised by the aggregation of α-synuclein in oligodendroglial cytoplasmatic inclusions.1 ...

Changes in the levels and activities of neurotrophic factors, such as brain-derived neurotrophic factor (BDNF), have been described in a number of neurodegenerative disorders, including Huntington ...

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 ...

For the roughly one million Americans living with Parkinson’s disease, every approved treatment does the same thing: manage symptoms. None slows the underlying brain degeneration. Results published in ...

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