Search

Pfrieger's Digest | Issue 12 - August 2024 – December 2024
Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C...
ANPDF
Jan 211 min read
36 views

Azafaros Community Update | January 9, 2025
Exciting Advances in Azafaros Clinical Programs for NPC and GM Gangliosidoses Azafaros has announced significant progress toward global...
ANPDF
Jan 101 min read
34 views

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...
ANPDF
Dec 20, 20241 min read
47 views

Meizon Innovations Community Update | November 5, 2024
Meizon has been granted both Orphan Drug Designation & Rare Pediatric Disease designation by the US FDA.
ANPDF
Nov 6, 20241 min read
168 views

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...
ANPDF
Oct 15, 20242 min read
9 views

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.
ANPDF
Sep 25, 20244 min read
111 views

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!
ANPDF
Sep 21, 20242 min read
110 views

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...
ANPDF
Sep 16, 20241 min read
12 views

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...
ANPDF
Sep 6, 20241 min read
15 views

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
ANPDF
Aug 7, 20241 min read
39 views

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...
ANPDF
Jul 17, 20241 min read
31 views

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...
ANPDF
Mar 27, 20241 min read
32 views

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...
ANPDF
Mar 5, 20241 min read
36 views

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...
ANPDF
Feb 8, 20241 min read
24 views

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...
ANPDF
Feb 3, 20241 min read
40 views

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...
ANPDF
Jan 10, 20241 min read
16 views

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...
ANPDF
Dec 28, 20231 min read
18 views

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...
ANPDF
Dec 21, 20231 min read
20 views

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...
ANPDF
Dec 20, 20231 min read
12 views

Meet and Greet with Cyclo Therapeutics
The Australian NPC Disease Foundation hosted a meet and greet with Cyclo Therapeutics on Zoom on 12th December.
ANPDF
Dec 13, 20231 min read
14 views