Rare Disease Report

National Lipid Association (NLA) Scientific Sessions 2015

LAL deficiency is a rare, progressive, disease that is most often diagnosed in childhood but in some cases, may not be diagnosed until adulthood.
Pradigastat may offer several benefits to patients with familial chylomicronemia syndrome (FCS).
Lysosomal acid lipase (LAL) deficiency is a rare, progressive, disease that is due to patients having an ineffective LAL. In this interview, Don Wilson, MD, FNLA, of Cook Children's Physician Network in Fort Worth, Texas describes this rare condition and the need for an approved treatment.
Eliot Brinton, MD, FAHA,FNLA, ABCL, director of the Utah Lipid Center in Salt Lake City, Utah lists the three signs that may indicate a child has lysosomal acid lipase (LAL) deficiency.
Patients with lysosomal acid lipase (LAL) deficiency appear to have higher 10-year Framingham cardiovascular risk than the general population. Sebelipase alfa (SA) treatment likely reduces that risk, based on an analysis of patients over 30 years of age in a poster presented at National Lipid Association (NLA) Scientific Sessions held in Chicago, Illinois this weekend.
Because the participants varied in their responses, the authors advised that patients be monitored individually when dosing lomitapide with these agents.
Patients with lysosomal acid lipase (LAL) deficiency treated with sebelipase alfa (SA) infusions for 20 weeks showed significant improvements in alanine aminotransferase (ALT) normalization and in a number of other important disease-related abnormalities.
Katelyn Phelps, DO, discusses a poster she presented at the National Lipid Association (NLA) Scientific Session in Chicago, Illinois that observed abnormal lipid and hemoglobin levels within a small pediatric population living in Iquitos, Peru.
Sandra Tremulis, president and founder of the Lipoprotein(a) Foundation, talks about the need for lipoprotein(a) to be routinely tested in patients to improve awareness, diagnosis, management, and research for patients with high lipoprotein(a).
Paula Soteropoulos, president and chief executive officer of Akcea Therapeutics, a lipid franchise subsidiary of Isis Pharmaceuticals talks about the phase 3 clinical studies the company is conducting to bring ISIS-APOCIIIrx to market.
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