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Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

From: Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies

Fig. 2

Kaplan–Meier Estimate of Survival, Birth to 60 Months. Kaplan–Meier estimates in VITAL and CL08 study participants compared with untreated infants with growth failure due to rapidly progressive LAL-D (natural history study LAL-1-NH01) [9]. The patient population in LAL-1-NH01 was similar in clinical characteristics to VITAL and CL08 patient populations. Adapted with permission from Orphanet Journal of Rare Diseases (Jones et al. [9]). LAL-D, lysosomal acid lipase deficiency

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172