Cyclo Therapeutics’ Trappsol Cyclo was able to induce clinical improvement in seven out of eight children with Neimann-Pick ...

In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients. IntraBio's ...

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

Accumulation of fat molecules is detrimental to the cell. Researchers have made a breakthrough in understanding how our cells manage to stay healthy by recycling important fat molecules. Their study ...

Eugen Mengel, discussing a significant clinical trial's efficacy results in treating Niemann-Pick disease type C (NPC). MIPLYFFA is approved for use alongside miglustat in patients aged two and ...

Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine), have made a breakthrough in ...

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

If cholesterol becomes trapped in a compartment within the cell, a disease called Niemann-Pick type C develops. This is a neurodegenerative disease that is also described as "childhood Alzheimer's." ...

Breakthrough NUS discovery sheds light on cellular recycling mechanism and potential for treating rare diseases Singapore ...