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Annual Severity Increment Score as a tool for stratifying patients with Niemann-Pick Disease Type C and for recruitment to clinical trials

Niemann-Pick disease type C (NPC) is a lysosomal storage disease with a heterogeneous neurodegenerative clinical course. Multiple therapies are in clinical trials and inclusion criteria are currently mainly based on age and neurological signs, not taking into consideration differential individual rates of disease progression.  This study evaluates a simple metric, denoted annual severity increment score (ASIS), that measures rate of disease progression for potential use in clinical practice.


08/17/2018 jjb

Amerigen and Dipharma Announce U.S. FDA Approval for Generic Miglustat 100 mg Capsules

NNPDF understands that there is great interest in the recent news of the FDA approved generic drug Miglustat for type 1 Gaucher disease. We are in touch with Amerigen Pharmaceuticals to find out more information on availability and will update this page as it becomes available.


UPDATE May 9, 2018

As way of further update regarding Miglustat, we have received the following information from Amerigen Pharmaceuticals: Miglustat capsules were approved on 4/17/2018 for monotherapy for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement is not a therapeutic option.  Miglustat is available through specialty pharmacies and wholesalers.  Patients who have questions on availability or pricing can contact Accredo,, the specialty pharmacy carrying the Amerigen product.

NNPDF Disclaimer:  The NNPDF does not engage in the practice of medicine. This information is provided only as an educational resource.  Individuals should contact their medical provider regarding appropriate treatment options for their specific conditions. NNPDF does not promote the use of any drug for families including those that are not FDA approved for NPC.  Miglustat to date has not been FDA approved for use in NPC nor has its efficacy been proven in the U.S. for any diseases other than type 1 Gaucher disease.

05/03/2018 jjb, 5/9/2018 cdk