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Table 2 Overview of the LSD disease registries sponsored by pharmaceutical industry

From: Registries for orphan drugs: generating evidence or marketing tools?

Disease Registry Year established Sponsor Patients enrolled (N)
LAL-D Lysosomal Acid Lipase (LAL) Deficiency Registry (ALX-LALD-501) 2012 Alexion 1000
Fabry Fabry Disease Registry 2001 Genzyme, a Sanofi Company 9000
  Fabry Outcome Survey (FOS) 2001 Shire 4000
Gaucher International Collaborative Gaucher Group (ICGG) Gaucher Registry 1991 Genzyme, a Sanofi Company 12,000
  Gaucher Disease Outcome Survey (GOS) 2010 Shire 1257
MPS I Mucopolysaccharidosis I (MPS I) Registry 2003 Genzyme, a Sanofi Company 1500
MPS II Hunter Outcome Survey (HOS) 2005 Shire 2000
MPS IVType A A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) 2014 BioMarin Pharmaceutical 583
MPS VI Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) 2005 BioMarin Pharmaceutical 200
MPS VII Mucopolysaccharidosis VII Disease Monitoring Program 2018 Ultragenyx Pharmaceutical Inc 35
Pompe Pompe Disease Registry 2004 Genzyme, a Sanofi Company 2000
  Alglucosidase Alfa Pompe Safety Sub-Registry 2015 Genzyme, a Sanofi Company 110
  Pompe Lactation Sub-Registry 2012 Genzyme, a Sanofi Company 5
  Pompe Pregnancy Sub-Registry 2011 Genzyme, a Sanofi Company 20
CLN2 Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease 2018 Jessica Scherr / Biomarin Pharmaceutical 30