Medically reviewed by Nicholas R. Metrus, MD 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 was considered a single disease until the 1960s, ... She is currently a lecturer at UC-Merced and co-wrote a children’s book with her son who lives with ASMD.

Zevra Therapeutics announces US commercial availability of Miplyffa™ (arimoclomol) for treatment of Niemann-Pick disease type C. News release. Zevra Therapeutics. November 21, 2024.

The FDA on Friday approved arimoclomol (Miplyffa) as the first treatment specifically for the genetic disorder Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative ...

'The Seventh Veil of Salome' by Silvia Moreno-Garcia is our 'GMA' Book Club pick for August. The novel is set in the 1950s during the Golden Age of Hollywood.

IntraBio claims FDA approval for its Aqneursa therapy for Niemann-Pick type C, the second new drug for the disease in four days. Skip to main content Monday 30 June 2025 . BlueSky ...

Today's biotech news updates include the dangers of off-brand GLP-1s, a foiled plan to privatize 23andMe, and Zevra's Niemann-Pick drug. Skip to Main Content RFK Jr.

Two new therapies just approved by the FDA have become the first ever treatments to slow the progression of Niemann-Pick Type C. Cindy predicts a cure is coming soon. “So, ...

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 were tested lacked neutralizing antibodies ...

Zevra Launches Niemann-Pick Disease Type C Drug Miplyffa In The U.S. November 22, 2024 — 03:26 am EST Written by RTTNews.com for RTTNews -> ...

Zevra Therapeutics drug Miplyffa is now FDA approved for Niemann-Pick disease type C, a lysosomal storage disorder. The affirmative regulatory decision took into account additional clinical data ...

Miplyffa is supplied as a capsule in 47mg, 62mg, 93mg, and 124mg dosage strengths.