A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who ...

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick ...

A new study in the journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 ...

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 ...

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

the global estimated prevalence of Niemann-Pick disease types A and B is around 1 in 2,50,000 individuals. In India, 32 patients, diagnosed with this condition by the Centres of Excellence (CoEs ...

Niemann-pick type C (NPC) is a rare neurological disorder where harmful levels of lipids (fats) accumulate in the body. Type A is the most serious form of the disorder, with infants succumbing to ...

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease ... adverse impacts on patients and families." ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

The following is a summary of “Serum chitotriosidase-1 (CHIT1) as candidate biomarker for mitochondriopathies,” published in ...

For Zevra Therapeutics, last year’s approval of the rare lysosomal storage disorder drug Miplyffa appears to be the gift that ...

Company on track for topline data from the 48-week interim analysis of 104 enrolled patients in TransportNPC ... Trappsol ® Cyclo™ for Niemann-Pick Disease Type C1 ("NPC1") at the 21 st ...