Nose picking could have serious health implications, new research has suggested, with scientists calling for further investigation into its potential link to Alzheimer’s disease. In a recent letter ...

Findings showed that among patients who also received miglustat, treatment with arimoclomol slowed NPC disease progression. The Food and Drug Administration (FDA) has approved Miplyffa ™ (arimoclomol) ...

*New research has raised concerns about nose picking and its potential link to Alzheimer’s disease. A recent letter in the American Journal of the Medical Sciences suggests that this habit might be a ...

Please provide your email address to receive an email when new articles are posted on . The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick ...

The submission includes data indicating a meaningful survival benefit for infantile-onset NPC patients treated with adrabetadex.

(RTTNews) - Zevra Therapeutics Inc. (ZVRA), a rare disease therapeutics company, Thursday, announced the commercial availability of Miplyffa for the treatment of Niemann-Pick disease type C along with ...

CELEBRATION, Fla., Oct. 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease therapeutics company, proudly supports the Niemann-Pick community on Global Niemann-Pick ...

LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients ...

CELEBRATION, Fla., Feb. 04, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with ...

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 ...