Mucopolysaccharidosis I Pipeline Dashboard, H1 2018

• The report provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis I.
• The report assesses Mucopolysaccharidosis I pipeline based on highest phase of development, type of sponsor, mechanism of action (MoA), route of administration (RoA), and molecule type.
• The report reviews Mucopolysaccharidosis I pipeline by companies and universities/research institutes based on information derived from company and industry-specific sources.
• The report covers pipeline products based on various stages of development for Mucopolysaccharidosis I ranging from discovery to pre-registration and undisclosed stages.

Sangamo BioSciences, BioMarin Pharmaceutical, Genzyme Corporation, Novazyme Pharmaceuticals, BioMarin Pharmaceutical, Amicus Therapeutics, Zacharon Pharmaceuticals, ArmaGen Technologies, Fondazione Telethon, GlaxoSmithKline, ReGenX Biosciences, OPKO Health, AngioChem, CRISPR Therapeutics, Inventiva Pharma, Immusoft Corporation, BioStrategies LC, Sangamo Therapeutics, PTC Therapeutics, Jupiter Orphan Therapeutics, Athersys, bene-Arzneimittel

Laronidase, NZ 1002, Alpha-L-iduronidase gene therapy, AGT 181, IVA 336, ELX 02, SB 318, Ataluren, Allogeneic stem cell therapy, Pentosan polysulfate sodium