Updates

INPDA Global Niemann Pick Disease Initiatives

INPDA Global Niemann Pick Disease Initiatives INPDA is actively engaged in a number of initiatives and campaigns which  have a wide-spread impact on a global scale . These include: Facilitating an update of the Consensus Clinical Management Guidelines for...

Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively

First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses. Both studies are part of Azafaros’ mission to develop treatments for the unmet need of patients with rare lysosomal storage disorders The...

AQNEURSA® (levacetylleucine) recommended for EU approval by the CHMP to treat Niemann-Pick Disease Type C

Zevra Submits Arimoclomol MAA to EMA for Niemann-Pick Disease Type C Treatment

Pfriegers Digest – 13th Issue

Pfriegers Digest – 13th Issue

Global Access Challenges for Niemann-Pick Therapies: Insights from an INPDA Community Survey

IntraBio Appoints Dr. Marc C. Patterson, US Chief Medical Officer

Update on PRONTO and Phase 2 RAINBOW study data with nizubaglustat, for patients with Niemann-Pick disease type C (NPC) GM1 and GM2 gangliosidoses.

Azafaros granted important regulatory designations and clearance by European authorities for global Phase 3 studies, to be initiated in 2025