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 ...

Cyclo Therapeutics’ Trappsol Cyclo was able to induce clinical improvement in seven out of eight children with Neimann-Pick ...

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 ...

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 ...

Guggenheim raised the firm’s price target on Zevra Therapeutics (ZVRA) to $21 from $20 and keeps a Buy rating on the shares. The firm, which is ...

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 ...