Lysosomal acid lipase deficiency

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At a Glance

Live from database

Regulatory Watchboard

2 events
Feb 2026Lysosomal Acid Lipase Deficiency in Risk Groups

AstraZeneca — NA

TrialRECRUITING
May 2013Lysosomal Acid Lipase (LAL) Deficiency Registry

Alexion Pharmaceuticals, Inc. — NA

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

1

Total programs

1

Open now

Patient Assistance Programs1

KANUMA

Alexion Pharmaceuticals, Inc.

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Approved Treatments

1 FDA-approved

KANUMA

(SEBELIPASE ALFA)Orphan drugstandard

Alexion Pharmaceuticals, Inc.

FDA label ↗

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Clinical Trial Landscape

2 active trials
2N/A
2Total recruiting
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Recent News & Research

1 article
Adv TherMar 24, 2026

Real-World Study of Management and Outcomes of Patients with Lysosomal Acid Lipase Deficiency (LAL-D) in France

Published in Adv Ther. Lacaille F et al.

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Specialist Network

Top 6 by expertise

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