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 ...

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.

aNeuroscience Department, The Cyprus Institute of Neurology and Genetics, 6 Iroon Avenue, 2371, Nicosia, Cyprus bCentre for Neuromuscular Disorders, The Cyprus Institute of Neurology and Genetics, 6 ...