Rare & Genetic Diseases

LIVE April 30th at 2:30 PM ET.
In this webinar, researchers will discuss how gene therapy has been used to treat disease and new research approaches to regenerative gene therapy.

EMA grants Orphan Drug designation to JR-171 for the treatment of mucopolysaccharidosis type I

Biobanking gives scientists access to thousands of biological samples, moving precision medicine one step closer to reality.

Results from cTAP indicate both could augment or replace placebo controls in DMD clinical trials

Preclinical data show injection of optimized AAV vector may correct gangliosidosis

Passage Bio plans Phase 1/2 trial of gene therapy in humans based on preclinical results

RNA-targeting gene therapy reverses molecular and functional features of DM1 in mice

Normal cell and tissue physiology is complex, even without pathologies that arise from myriad sources and impact multiple pathways. Rather than focusing on individual molecular entry points, phenotypic imaging enables a broader understanding of disease outcomes. This webinar will examine advances in imaging and high-content screening modalities to facilitate such understanding.