Hosted on MSN29d
An Overview of Niemann-Pick Disease
Niemann-Pick disease is a rare genetic medical condition. There are four variants of this disease, categorized as type A, type B, type C, and type D. Niemann-Pick disease causes a variety of ...
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 ...
pharmaphorum3d
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 ...
Cyclo Therapeutics Presents Positive Preliminary Data from Ongoing Phase 3 TransportNPC™ Open-Label Sub-Study in Patients (< 3 Years Old)
Cyclo Therapeutics, Inc. (Nasdaq: CYTH) ("Cyclo Therapeutics" or the "Company"), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation ...
ALZFORUM1mon
Microglia Drive Neurodegeneration in Niemann-Pick Type C
In 2017, neurologists at New York Presbyterian/ Columbia University’s movement clinic diagnosed Daniel with Niemann-Pick disease Type C (NPC): a lysosomal storage disorder caused by NPC1 mutations.