GlobalData on MSN8d
Cyclo Therapeutics logs efficacy win in pivotal Niemann-Pick disease trial
Cyclo Therapeutics’ Trappsol Cyclo was able to induce clinical improvement in seven out of eight children with Neimann-Pick ...
Medscape4d
Adult Niemann-Pick disease masquerading as sea blue histiocyte syndrome: report of a case confirmed by lipid analysis and enzyme assays.
Ultrastructural examination of these cells revealed lamellar myelin-like figures as described in Niemann-Pick (N-P) disease. Fibroblast enzyme assay studies and liver lipid analyses performed ...
pharmaphorum4mon
FDA clears first drug for Niemann-Pick disease
Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease. The US regulator has cleared Miplyffa (arimoclomol ...
pharmaphorum4mon
Second Niemann-Pick approval brings more hope to patients
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 ...