Wolman Disease News and Research

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Kanuma approved as first treatment for patients with LAL deficiency

Today, the U.S. Food and Drug Administration approved Kanuma (sebelipase alfa) as the first treatment for patients with a rare disease known as lysosomal acid lipase (LAL) deficiency.

8 Dec 2015

Synageva announces publication of LAL Deficiency review in Atherosclerosis

Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare diseases, today announced the publication of an overview of lysosomal acid lipase deficiency (LAL Deficiency) in the online version and an upcoming print edition of Atherosclerosis, the official journal of the European Atherosclerosis Society.

21 Apr 2014

Synageva BioPharma presents data on cholesteryl ester storage disease at NLA annual meeting

Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare diseases, announced three poster presentations at the National Lipid Association annual meeting held in Las Vegas, Nevada, May 30- June 2, 2013, as well as a Synageva-sponsored satellite symposium held on Friday, May 31, 2013.

1 Jun 2013

FDA designates Synageva's sebelipase alfa as Breakthrough Therapy for early onset LAL Deficiency

Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare diseases, today announced that the U.S. Food and Drug Administration granted Breakthrough Therapy designation to sebelipase alfa for the treatment of early onset lysosomal acid lipase deficiency, also known as Wolman disease.

20 May 2013

Synageva BioPharma reports study results of sebelipase alfa in adults with late onset LAL Deficiency

Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare diseases, today reported 12-month results from an ongoing extension study with sebelipase alfa in adults with late onset Lysosomal Acid Lipase Deficiency.

9 May 2013

Synageva announces 12-week data from sebelipase alfa Phase I/II study on LAL Deficiency

Synageva BioPharma Corp. (Synageva), a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced publication of the 12-week Phase I/II extension study data of sebelipase alfa in adults with lysosomal acid lipase deficiency (LAL Deficiency) in the online version and an upcoming print edition of Hepatology

5 Feb 2013

FDA grants fast track designation to Synageva for SBC-102 program for Lysosomal Acid Lipase Deficiency

Synageva BioPharma Corp., a privately held biopharmaceutical company developing therapeutic products for rare disorders, announced today that their lead program, SBC-102, an enzyme replacement therapy for Lysosomal Acid Lipase (LAL) Deficiency, currently in clinical trials, has been granted fast track designation by the U.S. Food and Drug Administration (FDA).

23 Jun 2011

Trimeris, Synageva enter definitive merger agreement

Synageva BioPharma Corp., a privately held biopharmaceutical company developing therapeutic products for rare disorders, and Trimeris, Inc., announced today that they have entered into a definitive agreement under which Synageva will merge with Trimeris in an all-stock transaction.

13 Jun 2011

Synageva supports Fourth International Rare Disease Day

Synageva BioPharma Corp., a privately held biopharmaceutical company that is developing therapeutic products for rare disorders, announced today that the company is supporting the Fourth International Rare Disease Day observance on February 28, 2011.

28 Feb 2011

Synageva's SBC-102 enzyme replacement therapy for LAL deficiency granted European orphan product designation

Synageva BioPharma Corp., a privately held biopharmaceutical company, announced that the European Medicines Agency (EMA) granted orphan product designation for SBC-102 (Recombinant Human Lysosomal Acid Lipase), the Company's enzyme replacement therapy in development to treat Lysosomal Acid Lipase (LAL) Deficiency.

10 Nov 2010

FDA grants orphan drug designation for Synageva BioPharma's SBC-102

Synageva BioPharma Corp., a privately held biopharmaceutical company, announced that the U.S. Food and Drug Administration has granted orphan drug designation for SBC-102, the Company's enzyme replacement therapy in development to treat Lysosomal Acid Lipase Deficiency, also known as Wolman Disease and Cholesteryl Ester Storage Disease, a condition for which there is currently no approved treatment.

7 Jul 2010

Synageva BioPharma seeks FDA orphan drug designation for SBC-102 enzyme replacement therapy

Synageva BioPharma Corp., a privately held biopharmaceutical company, today announced its filing with the U.S. Food and Drug Administration (FDA) requesting orphan drug designation for its program, SBC-102. This program is an enzyme replacement therapy in preclinical development to treat lysosomal acid lipase (LAL) deficiency, also known as Wolman disease or cholesteryl ester storage disease (CESD), a condition for which there is currently no effective treatment.

4 May 2010