Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for the bleeding condition. The pioneering gene therapy developer will be ...

The treatment, called MCO-010 (sonpiretigene isteparvovec), comprises a gene that codes for the ambient ... 100,000 people in the US and an estimated 2 million people worldwide suffer from RP ...

Feb. 25, 2025 — Investigators have conducted a multi-ancestry, whole genome sequencing association study of Alzheimer's disease and found evidence for 16 new susceptibility genes, expanding the ...

In neuronal cells, the regulation of RNA is crucial for the spatiotemporal control of gene expression, but how the correct localization, levels, and function of synaptic proteins are achieved is not ...