Search


Cyclo Therapeutics Community Update | June 18, 2025
June 18, 2025 – Newark, NJ Rafael Holdings has announced that an independent Data Monitoring Committee (DMC) has completed its scheduled...

ANPDF
Jun 202 min read


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


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


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


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


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)...

ANPDF
Oct 23, 20241 min read


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 14, 20242 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.

ANPDF
Sep 25, 20244 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!

ANPDF
Sep 21, 20242 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...

ANPDF
Sep 16, 20241 min read


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


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


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


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...

ANPDF
Jun 1, 20241 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...

ANPDF
Mar 27, 20241 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.

ANPDF
Mar 14, 20241 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...

ANPDF
Mar 5, 20241 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...

ANPDF
Feb 8, 20241 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...

ANPDF
Feb 3, 20241 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...

ANPDF
Feb 1, 20241 min read