Nitrated α-synuclein is upregulated in the CSF of patients with Parkinson’s Disease (PD), Lewy body dementia (DLB), multiple system atrophy (MSA), and other synucleinopathies.

Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease originating from biallelic pathogenic variants in the ARSA gene, mainly affecting young children.

Writing in Molecular Therapy Nucleic Acids, researchers hypothesized that using poly(A) tail mimetics to enhance mRNA expression from haploinsufficiency-associated genes could be a disease-modifying treatment strategy.

Neuroendocrine prostate cancer (NEPC) is the most aggressive form of prostate cancer, responsible for up to 20% of therapy-resistant cases and associated with rapid disease progression. It is characterized by a loss of typical androgen receptor signaling, conferring antiandrogen therapy resistance, and abnormal expression of neuroendocrine markers, along with small cell morphology.

NLRP3 inflammasome inhibitors are described in a Zomagen Biosciences Ltd. patent and are reported to be useful for the treatment of autoimmune, cardiovascular, inflammatory, liver, lung, metabolic and neurological disorders.