TABLE 3.

Subject characteristics

	Evaluable patients (n=43)
Gender, n1
Male	21
Female	22
Age at enrollment, n
2 to <10 years	21
10 to <20 years	13
20 to <30 years	4
30 to < 40 years	2
>40 years	3
Prior steroid response, n
Had a prior response	19
Never responded	17
Unknown	7
Genotypes (n)
Complete response to L-leucine	RPL35A (1), Unknown (1)
Partial response to L-leucine	RPS17 (1), RPS24 (1), Unknown (3)
No response to L-leucine	RPS19 (11), RPS17 (4), RPS24 (2), RPS26 (1), RPS29 (1), RPL5 (1), RPL11 (2), RPL35A (2), Unknown (12)

¹n, number of subjects