Clinical Trials

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

Mandos Health Media Release | October 22, 2024

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Oct 231 min read

Mandos Health Media Release | October 22, 2024

Read the letter from Mandos Health here: The Mandos Health October 2024 update highlights their progress in Niemann-Pick Type C (NPC)...

44 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...

12 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

39 views

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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

Cyclo Therapeutics Media Release | May 30, 2024

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Jun 11 min read

Cyclo Therapeutics Media Release | May 30, 2024

Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of...

41 views

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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

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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.

20 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...

24 views

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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

IntraBio Community Update | February 1, 2024

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Feb 11 min read

IntraBio Community Update | February 1, 2024

New Drug Application for IB1001 for the treatment of NP-C disease was submitted to the US FDA in January 2024 Read the full statement...

50 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

NP-C Corner: Will I be a lab rat? An exploration of clinical trials in the world of Niemann-Pick.

Ellie Van Velsen

Jan 83 min read

NP-C Corner: Will I be a lab rat? An exploration of clinical trials in the world of Niemann-Pick.

Ellie explores what clinical trials are and how they impact patients of Niemann-Pick diseases.

62 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

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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.

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