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 ...

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 ...

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 ...

It never occurred to the family that both brothers could have the same rare genetic condition. In 2017, neurologists at New York Presbyterian/ Columbia University’s movement clinic diagnosed Daniel ...

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 ...

Eugen Mengel, discussing a significant clinical trial's efficacy results in treating Niemann-Pick disease type C (NPC). MIPLYFFA is approved for use alongside miglustat in patients aged two and ...

Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposium Eight abstracts discussing data and clinical experience associated with MIPLYFFA accepted for poster ...

Oragenics (NYSE American: OGEN) announced that its Chief Medical Officer, Dr. James P. Kelly, will be a featured panelist at the 12th Annual Brain He ...