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)...
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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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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.
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ANPDF
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!
101 views
ANPDF
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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ANPDF
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
36 views
ANPDF
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...
23 views
ANPDF
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
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
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.
18 views
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...
34 views
ANPDF
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
ANPDF
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...
39 views
ANPDF
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
ANPDF
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...
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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.
61 views
ANPDF
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...
14 views
ANPDF
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
ANPDF
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
ANPDF
Jun 29, 20231 min read
Intrabio Media Release | June 29, 2023
"IntraBio Announces Positive Pivotal Trial Results of IB1001 for the Treatment of Niemann-Pick Disease Type C" Read the full article...
47 views
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