Meizon Innovations Community Update | November 5, 2024
Meizon has been granted both Orphan Drug Designation & Rare Pediatric Disease designation by the US FDA.
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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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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)...
38 views
ANPDF
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.
88 views
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!
103 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...
12 views
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
38 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...
35 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...
15 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
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