Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

Author:

Gaudet D,Méthot J,Déry S,Brisson D,Essiembre C,Tremblay G,Tremblay K,de Wal J,Twisk J,van den Bulk N,Sier-Ferreira V,van Deventer S

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference39 articles.

1. Brunzell JD, Deeb SS . Familial lipoprotein lipase deficiency, Apo C-II deficiency and hepatic lipase deficiency. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds). The Metabolic Basis of Inherited Disease 8th ed. McGraw-Hill: New York, NY, 2001, pp 2789–2816.

2. Black DM, Sprecher DL . Dietary treatment and growth of hyperchylomicronemic children severely restricted in dietary fat. Am J Dis Child 1993; 147: 60–62.

3. Chait A, Robertson HT, Brunzell JD . Chylomicronemia syndrome in diabetes mellitus. Diabetes Care 1981; 4: 343–348.

4. Chait A, Brunzell JD . Chylomicronemia syndrome. Adv Int Med 1992; 37: 249–273.

5. Santamarina-Fojo S . The familial chylomicronemia syndrome. Endocrinol Metab Clin North Am 1998; 27: 551–567.

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