Quantitative systems pharmacology (QSP) analysis predicts that olipudase alfa treatment of patients with extreme acid sphingomyelinase deficiencies can result in meaningful debulking of visceral tissue sphingomyelin

Fluorinated biomaterials have interesting properties attractive for their application in several biomaterials and biotechnology niches. Biomedical grade solids and liquids are commercially available. These biomaterials are central to vascular grafts and hemodialysis access, hernia repair, and medical tubing essential to catheters. Intrinsically expensive materials, fluoropolymers have been recognized for their chemical stability and unique interfacial properties with biological fluids and cellular components. Fluorinated materials, coatings, and films all interact strongly with biological milieu proteins, creating adherent protein layers that govern further biomaterials performance. This property is exploited in biotechnology of blotting membranes and in preclotted cardiovascular implants. Perfluoropolymers have difficult processing challenges, but unique specialized fabrication methods exploit qualities in certain fluorinated materials, including bulk mechanical processing for creation of microporous perfluoropolymer meshes and fabrics, and high-precision size-tolerance in making fluorinated medical tubing and multilumen catheters. Perfluorofluids have limited volatility, high density, high gas solubility, insolubility with aqueous milieu and emulsified stability, and low toxicity in human tissue, which are attractive characteristics for imaging and oxygen/drug delivery capacities. Fluorinated solids are not generally mechanically useful because of their tendency to cold flow under shear stress. In addition to chemical and physical characteristics, numerous biomedical applications for fluorinated materials are described.