The selected projects demonstrated significant potential to address unmet needs within these rare communities. The recipients include: Fabry disease: Mitra Tavakoli, University of Exeter, Exeter, UK, ...

This AJMC ® Profiles in Care focuses on payer challenges and strategies involved when managing treatments for lysosomal storage diseases and other rare diseases. Stay ahead of policy, cost, and ...

The right approaches are demonstrating that it is possible to breach the defensively designed blood-brain barrier despite its ...

A Denali treatment for a rare lysosomal storage disorder, Hunter syndrome, has posted encouraging biomarker results in a small, open-label Phase 1/2 trial. The genetic disorder is caused by mutations ...

Lysosomal storage diseases (LSDs) are rare, inherited disorders that often take years to diagnose due to their complex and varied symptoms. Recent case studies and registry data are helping doctors ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced the presentation of four ...

LONDON--(BUSINESS WIRE)--Technavio’s latest market research report on the global lysosomal storage diseases (LSDs) market provides an analysis of the most important trends expected to impact the ...

The lysosome is a critical site for maintaining cellular catabolic function, ionic balance, nutrition sensing, metabolic balance and immunity balance. DC, dendritic cell. A new study published in ...

Macrophages are the specialized phagocytic cells that respond to toxic particle depositions to exert inflammatory and clearance responses. These long-lived immune cells engulf foreign substances like ...

Stanford researchers constructed an atlas of the proteins found in the lysosome across different brain cell types, a recently published paper reported. Lysosomes are cell organelles that play ...