The cause of Niemann-Pick disease is relatively complicated. People who have this disease inherit one of several genetic defects, resulting in a buildup of sphingomyelin, a type of fat. As the ...

Niemann-Pick type B is caused by a defect in the SMPD1 gene, which results in reduced production of Acid Sphingomyelinase, which causes sphingomyelin to build up in the cells, which in turn ...

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 ...

Cyclo Therapeutics, Inc. ("Cyclo Therapeutics" or the "Company"), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and ...

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

Using advanced cell imaging techniques and computer ... 6, 2024 — Scientists report on new insights into the mechanisms of 'Niemann-Pick type C' (NPC), a rare neurodegenerative disease ...

Hans Niemann has challenged Arjun Erigaisi and Fabiano Caruana for matches but both are yet to accept the challenge. Image: X / @FIDE_chess American grandmaster Hans Niemann believes that the ...

Feb. 4, 2025 — Research has shown that a potential new targeted therapy for childhood brain cancer is effective in infiltrating and killing tumor cells in preclinical models tested in mice.

CD36 is expressed in multiple cell types, including platelets, monocytes/macrophages ... be driven by the concentration gradient or facilitated by other proteins such as CD36. Cholesterol uptake is ...

To enter a human cell, an ebolavirus glycoprotein has to ... helped discover the cholesterol-trafficking protein Niemann-Pick C1 (NPC1) as this critical Ebola entry receptor.

Torque GC captain Joaquin Niemann will look to follow up on last year's success in Saudi Arabia when LIV Golf opens the season under the lights at Riyadh Golf Club on Thursday. The 26-year-old ...