Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

Table 1 Baseline characteristics

Patient ID	Age (years)	eGFR (mL/min/1.73 m²)	24-hour protein (mg)	Left Ventricle Mass (g)	*GLA* mutation	HEK assay: amenable mutation?
2-0102	27	143.8	270	138	p.L415P	NO
2-0103	23	127.3	147	228	p.P259R	YES
2-0104	18	156.1	131	123	p.P259R	YES
2-0202	65	33	4640	312	p.R301Q	YES
3-0301	39	121.1	--^a	225	p.F295C	YES ^b
3-0302	31	134.5	--^a	144	p.C94S	NO
3-0303	36	90.3	--^a	175	p.R112C	NO
3-RF01	55	92.5	<100	231	p.N215S	YES
3-RFO3	47	135.7	150	200	p.P205T	YES

^a Value not available.
^b Mutation was amenable only when higher doses of migalastat hydrochloride were used.

ISSN: 1750-1172