Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

Research programme: Niemann-Pick disease type B gene therapy - Sanofi Genzyme

Drug Profile

Research programme: Niemann-Pick disease type B gene therapy - Sanofi Genzyme

Latest Information Update: 16 Dec 2016

Price : $50 *
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

  • Originator Genzyme Corporation
  • Developer Genzyme Corporation; Mount Sinai School of Medicine
  • Class Gene therapies
  • Mechanism of Action Gene transference; Sphingomyelin phosphodiesterase stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Niemann-Pick disease type B

Most Recent Events

  • 26 Feb 2009 Discontinued - Preclinical for Niemann-Pick disease type B in USA (unspecified route)
  • 22 Jun 2005 Data presented at the 8th Annual Meeting of the American Society of Gene Therapy (ASGT-2005) have been added to the Metabolic Disorders pharmacodynamics section
  • 29 Jul 2003 Genzyme General, Genzyme Biosurgery and Genzyme Molecular Oncology are now called Genzyme Corporation
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top