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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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Azafaros Community Update | December 20, 2024

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4 days ago1 min read

Azafaros Community Update | December 20, 2024

Exciting Advances in Azafaros Clinical Programs for NPC and GM Gangliosidoses Today, Azafaros shared an important update about their...

43 views

Meizon Innovations Community Update | November 5, 2024

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Nov 61 min read

Meizon Innovations Community Update | November 5, 2024

Meizon has been granted both Orphan Drug Designation & Rare Pediatric Disease designation by the US FDA.

113 views

Exciting News: New INPDR Site Opening in Melbourne for Niemann-Pick Disease

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Oct 152 min read

Exciting News: New INPDR Site Opening in Melbourne for Niemann-Pick Disease

15 October 2024 - INPDR, Tyne & Wear England. We are thrilled to share the announcement of a significant development in the fight against...

8 views

Second treatment approved by FDA for Niemann-Pick disease, type C: Introducing AQNEURSA

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Sep 254 min read

Second treatment approved by FDA for Niemann-Pick disease, type C: Introducing AQNEURSA

FDA approval of AQNEURSA offers new hope for Australian families with Niemann-Pick disease, promising better treatment and quality of life.

94 views

A New Hope for NP-C Families: Introducing Miplyffa

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Sep 212 min read

A New Hope for NP-C Families: Introducing Miplyffa

New Hope for NP-C Families: Miplyffa, the first FDA-approved treatment for NPC, offers a promising future for our loved ones. Stay informed!

105 views

Mandos Health Media Release | September 10, 2024

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Sep 161 min read

Mandos Health Media Release | September 10, 2024

Read the letter from Mandos Health here: Here is a summary of the article: INPDA 2023 Meeting : Mandos attended the 2023 INPDA meeting in...

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Pfrieger's Digest | Issue 11 - March 2024 – July 2024

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Sep 61 min read

Pfrieger's Digest | Issue 11 - March 2024 – July 2024

Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C...

15 views

Zevra Therapeutics Media Release | August 2, 2024

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Aug 71 min read

Zevra Therapeutics Media Release | August 2, 2024

FDA Advisory Committee Votes Favourably that the Data Support Arimoclomol as Effective Treatment for Patients with NP-C

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Jul 171 min read

Azafaros Media Release | July 16, 2024

Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C Read the...

24 views

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...

31 views

Zevra Therapeutics Media Release | March 4, 2024

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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...

36 views

IntraBio Media Release | February 7, 2024

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Feb 81 min read

IntraBio Media Release | February 7, 2024

The New England Journal of Medicine has published a "Science behind the study" editorial on IB1001 Read the full announcement from...

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Feb 31 min read

IntraBio Media Release | February 7, 2024

The New England Journal of Medicine has published the detailed results of the IB1001-301 Phase 3, Pivotal study with N-acetyl-L-leucine...

40 views

Zevra Therapeutics Media Release | January 8, 2024

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Jan 101 min read

Zevra Therapeutics Media Release | January 8, 2024

Zevra Therapeutics Receives FDA Acceptance Of Resubmission Of NDA For Arimoclomol As A Treatment For Niemann-Pick Disease Type C Read the...

16 views

Zevra Therapeutics Media Release | December 27, 2023

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Dec 28, 20231 min read

Zevra Therapeutics Media Release | December 27, 2023

Zevra Therapeutics Announces Resubmission Of Arimoclomol New Drug Application To The U.S. Food And Drug Administration Read the full...

18 views

Azafaros Media Release | December 11, 2023

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Dec 21, 20231 min read

Azafaros Media Release | December 11, 2023

Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled Read the full statement from...

20 views

Cyclo Therapeutics Media Release | December 18, 2023

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Dec 20, 20231 min read

Cyclo Therapeutics Media Release | December 18, 2023

Cyclo Therapeutics Announces Positive Outcome from Type C Meeting with the U.S. FDA Discussing Trappsol® Cyclo™ Clinical Program for the...

10 views

ANPDF

Dec 13, 20231 min read

Meet and Greet with Cyclo Therapeutics

The Australian NPC Disease Foundation hosted a meet and greet with Cyclo Therapeutics on Zoom on 12th December.

14 views

Zevra Therapeutics Media Release | November 20, 2023

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Nov 21, 20231 min read

Zevra Therapeutics Media Release | November 20, 2023

Zevra announces the completion of its acquisition of Acer Therapeutics. Zevra states they are committed to the arimoclomol expanded...

44 views

Kisbee Therapeutics and Zevra Therapeutics Updates

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Sep 11, 20231 min read

Kisbee Therapeutics and Zevra Therapeutics Updates

"We remain steadfast in our key priorities to the NPC community," - Zevra Therapeutics Read the full statement from Zevra Therapeutics...

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