Read the full Community Update from Mandos Health here: Mandos Health has shared an October update highlighting exciting progress in Niemann-Pick type C (NPC) research. Their investigational therapy, adrabetadex , continues to show promise, with new data suggesting a substantial survival benefit  in infantile-onset NPC1 and evidence of improved cholesterol trafficking . Their survival study was recognised as an “Abstract of Distinction”  at the American Neurological Associati

Dr Ya Hui Hung presented at the 2025 INPDA Bi-Annual Meeting. Australia is emerging as a hub of innovation in rare disease research, and...

DL33, developed by Meizon Innovations, is preparing for the next stage of testing — and could one day change the future of childhood dementia. 💜

Xenpozyme® (olipudase alfa) has been listed on the Life Saving Drugs Program (LSDP) for patients with Acid Sphingomyelinase Deficiency (ASMD).

Frank W. Pfrieger, PhD Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD)...

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

Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C...

Exciting Advances in Azafaros Clinical Programs for NPC and GM Gangliosidoses Azafaros has announced significant progress toward global...

Exciting Advances in Azafaros Clinical Programs for NPC and GM Gangliosidoses Today, Azafaros shared an important update about their...

Meizon has been granted both Orphan Drug Designation & Rare Pediatric Disease designation by the US FDA.

Read the letter from Mandos Health here: The Mandos Health October 2024 update highlights their progress in Niemann-Pick Type C (NPC)...

15 October 2024 - INPDR, Tyne & Wear England. We are thrilled to share the announcement of a significant development in the fight against...

FDA approval of AQNEURSA offers new hope for Australian families with Niemann-Pick disease, promising better treatment and quality of life.

New Hope for NP-C Families: Miplyffa, the first FDA-approved treatment for NPC, offers a promising future for our loved ones. Stay informed!

Read the letter from Mandos Health here: Here is a summary of the article: INPDA 2023 Meeting : Mandos attended the 2023 INPDA meeting in...

Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C...

FDA Advisory Committee Votes Favourably that the Data Support Arimoclomol as Effective Treatment for Patients with NP-C

Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C Read the...

Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of...

IntraBio Announces U.S. FDA Accepts New Drug Application for IB1001 for the Treatment of Niemann-Pick disease type C Read the full...