National Lysosomal Acid Lipase Deficiency Study

计划入组 810 人 · 首次发布于 2015 年 2 月 26 日

Screening for Lysosomal Acid Lipase Deficiency

计划入组 22 人 · 首次发布于 2016 年 10 月 6 日

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

计划入组 60 人 · 首次发布于 2012 年 10 月 30 日

Sebelipase alfa(Kanuma)

Rx

Recombinant human lysosomal acid lipase(Shire Human Genetic Therapies Inc)

Liprotamase lipase

Shanghai Ajinomoto Amino Acid Co., Ltd.

China

LYSOSOMAL AND RARE DISORDERS RESEARCH AND TREATMENT CENTER, INC.

United States

Rockwell Medical

United States

NICE Rejects Alexion's Kanuma for Ultra-Rare LAL-D Treatment, Citing High Costs

Pricing Dynamics of Rare Disease Drugs: How Prevalence and Patient Numbers Shape Market Access

Milky-White Blood in Infant Reveals Ultra-Rare Familial Lipoprotein Lipase Deficiency