An observational disease and clinical outcomes registry of patients with lysosomal acid lipase (LAL) deficiency (ALX-LALD-501)

Documents

Study protocol

Initial protocol

riskmgtsystem redacted-annex-6-alx-lald-501-3.pdf

Study results

Study report

Other information

Publications

Study publications

Francis M, Balwani M, Balistreri W, D’Antiga L, Fang S, Jones S, Ros E, Abel F,…