March - 2024

Community News and Events

Welcome to the ANPDF blog, where we share the latest updates, stories, and breakthroughs in the fight against Niemann-Pick Disease. Join us as we raise awareness, support families, and drive research forward.

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ANPDF

Mar 271 min read

IntraBio Media Release | March 26, 2024

IntraBio Announces U.S. FDA Accepts New Drug Application for IB1001 for the Treatment of Niemann-Pick disease type C Read the full...

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ANPDF

Mar 141 min read

Azafaros Media Release | March 12, 2024

Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients.

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In the media: Vertigo drug could be used to treat Niemann-Pick type C disease, known as ‘childhood Alzheimer’s’

ANPDF

Mar 102 min read

In the media: Vertigo drug could be used to treat Niemann-Pick type C disease, known as ‘childhood Alzheimer’s’

By Sarah Booth (Herald Sun), March 10, 2024. Read the full article here. Melbourne, Victoria – In a significant medical breakthrough,...

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Zevra Therapeutics Media Release | March 4, 2024

ANPDF

Mar 51 min read

Zevra Therapeutics Media Release | March 4, 2024

Zevra Therapeutics Provides FDA Update On The PDUFA Action Date For Arimoclomol As A Treatment For Niemann-Pick Disease Type C Read the...

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