Rare lysosomal disease registries: lessons learned over three decades of real-world evidence

Table 2 Total number of patients and person-years in the Rare Disease Registries

Registry	Year Registry was established	Current data			Person-years
		Total countries*	Total Registry sites**	Total patients	Total person-years from birth to last follow-up†		Total person-years from diagnosis to last follow-up‡		Total person-years from treatment initiation to last follow-up§
		Total countries*	Total Registry sites**	Total patients	Person-years	N	Person-years	N	Person-years	N
ICGG Gaucher Registry	1991	64	278	6872	266,543	6844	112,115	6481	67,470	5595
Fabry Registry	2001	47	243	7930	344,445	7897	78,220	7267	38,523	5017
MPS I Registry	2003	41	144	1325	18,598	1323	13,497	1297	10,086	1176
Pompe Registry	2004	47	240	2467	87,251	2463	21,761	2405	13,510	2210
Total			805	18,594	716,837	18,527	235,593	17,450	129,589	13,798

All Data as of February 2022
*Includes currently and historically active countries/regions
**Includes currently active sites where at least one patient is enrolled
^†Data are shown for patients with non-missing dates of birth and last follow-up
^‡Data are shown for patients with non-missing dates of diagnosis and last follow-up
^§Data are shown for ever-treated patients with non-missing dates of treatment initiation and last follow-up
MPS I, Mucopolysaccharidosis type I

ISSN: 1750-1172