Updates

IntraBio Receives European Commission Approval of AQNEURSA® for the Treatment of Niemann-Pick Type C Disease

AUSTIN, Texas–(BUSINESS WIRE)–IntraBio Inc. today announced that the European Commission granted marketing authorization to AQNEURSA® (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick Type C (NPC) disease, following a positive opinion from the Committee for Medicinal Products for Human...

Patient Information Sheet: NAVIGATE Phase 3 Clinical Trial

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