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 ...

which causes sphingomyelin to build up in the cells, which in turn interferes with the function of several organs in the body. All of the types of Niemann-Pick disease are autosomal recessive ...

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 ...

To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1‐800-FDA-1088 or .

Takeda’s agreement with Evox covers a preclinical-stage candidate for Niemann-Pick disease type C (NPC ... Vertex's gene-editing therapy for sickle cell disease, Casgevy, has been given ...

This suggests that restoration of SMPD1 deficiency could be a viable interception point and therapeutic strategy for Parkinson’s disease. Such a concept is currently under trial for Niemann-Pick ...

Using advanced cell imaging techniques and ... on new insights into the mechanisms of 'Niemann-Pick type C' (NPC), a rare neurodegenerative disease associated with dementia that can manifest ...

5, 2024 — Stem cell transplantation is used to treat several types of blood cancers, but carries the risk of a life-threatening side effect called graft-versus-host disease. Results from a ...

Bone-marrow-derived multipotent progenitor stromal cells — or mesenchymal stem cells (MSCs) — are the common predecessors of cells of the mesenchymal lineage, such as bone, cartilage and fat ...