The protein Spns1 helps transport fat molecules out of lysosomes, preventing harmful buildup and offering insights for rare ...

Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National ...

New research has made a breakthrough in understanding how our cells manage to stay healthy by recycling important fat ...

WORLD Symposium 2025 is an international conference or lysosomal storage disorders experts to share and exchange insights for researching better treatment of the disorders. At thi ...

AceLink is advancing a small-molecule treatment option for Fabry disease, which could provide a more convenient alternative ...

Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions ...

Dr. Lee will oversee the clinical development of Lycia’s lead programs, LCA-0061 for IgE-mediated diseases and LCA-0321 for ...

Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.

Fabry disease is an X-linked lysosomal storage disorder caused by a deficiency of the enzyme α-galactosidase A (GLA), which leads to complex multisystemic involvement, with the majority of patients ...

Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposiumEight abstracts discussing data and clinical ...