Alpha-1 Antitrypsin Deficiency (AATD) is a rare genetic disorder caused by SERPINA1 gene mutations, leading to conditions like pulmonary emphysema and liver dysfunction. The AATD market is in its ...

4D Molecular Therapeutics trades below its cash balance, offering significant upside as its late-stage gene therapy programs advance. FDMT's lead asset, 4D-150, targets wet AMD and DME, with Phase 3 ...

“Taken together, our findings highlight both brain region-specific vulnerabilities and global molecular perturbations associated with alpha-synuclein biology and provide insights into early ...

UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...

Thanks to groundbreaking in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a ...