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 |
| Â | Fabry Outcome Survey (FOS) | 2001 | Shire | 4000 |
Gaucher | International Collaborative Gaucher Group (ICGG) Gaucher Registry | 1991 | Genzyme, a Sanofi Company | 12,000 |
| Â | 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 |
| Â | Alglucosidase Alfa Pompe Safety Sub-Registry | 2015 | Genzyme, a Sanofi Company | 110 |
| Â | Pompe Lactation Sub-Registry | 2012 | Genzyme, a Sanofi Company | 5 |
| Â | 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 |