Search
ANPDF
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
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...
20 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...
12 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
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...
49 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
ANPDF
Jan 10, 20231 min read
Azafaros Media Releases | January 5-9, 2023
"Azafaros Receives FDA's IND Clearance and Fast Track Designation for Lead Asset AZ-3102" Read the press release from January 5, 2023...
44 views
ANPDF
Dec 6, 20221 min read
Intrabio Media Release | December 5, 2022
"IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on...
32 views
ANPDF
Oct 10, 20221 min read
Pfrieger's Digest | Issue 7 - September 2022
Pfrieger's Digest for Niemann-Pick type C: Summaries of research advances on selected peer-reviewed publications in scientific journals....
41 views
ANPDF
Jun 30, 20222 min read
It's a wrap! 2022 NPC Conference
The annual NPC Conference, both live and online, attended by patients, families, clinicians and researchers of Niemann-Pick disease.
127 views
ANPDF
Jun 6, 20221 min read
Pfrieger's Digest | Issue 6 - May 2022
Pfrieger's Digest for Niemann-Pick type C: Summaries of research advances on selected peer-reviewed publications in scientific journals....
26 views
ANPDF
Mar 25, 20221 min read
Azafaros Media Release | March 24, 2022
"Second Orphan Drug Designation Supports Azafaros’ Strategy to Develop AZ-3102 as a Disease Modifying Treatment in a Range of Severe Rare...
22 views
ANPDF
Mar 25, 20221 min read
Orphazyme Media Release | March 22, 2022
"Orphazyme announces withdrawal of European Marketing Authorisation Application for arimoclomol for the treatment of Niemann-Pick disease...
29 views
ANPDF
Jan 29, 20221 min read
New Research Article | January 26, 2022
New research article published: "Severely impaired CTL killing is a feature of the neurological disorder
Niemann-Pick Syndrome type C1"
107 views
ANPDF
Oct 11, 20211 min read
Niemann-Pick Disease Type C: Working together to make a difference
This lecture will present the Australian collaboration and latest research on NP-C, a rare genetic disorder that leads to childhood...
43 views
ANPDF
Jul 27, 20211 min read
Reconnecting at the 2021 NPC Conference
Adapting to the times, our Australian NPC community had the opportunity to connect at this year's conference with our first-ever...
11 views