LAL Deficiency (Lysosomal Acid Lipase Disease) & Wolman Disease patient support
My daughter is 4 1/2 years old have LAL D. The diagnosis took 18 months to arrive :-(
Nowadays she is in a clinical trial since november 2014 in Spain.
Now she has a normal liver size, and good cholesterol but not yet the liver enzymes and she has scarring on his liver.
During the two-hour infusion she is fine (a little bit bored). When it ends she leaves happy the hospital.
Right now we are waiting for negotiations of Alexion and the Spanish Government so that patients have access to treatment.
There are two kids 10 year old who are not in the clinical trial and they are waiting several months the approval to treatment before the Kanuma be marketed.
In January 2015 my husband and I decided to create the LAL D Spanish Organization (www.aelald.org) because did not exist in Europe.
We try to act so all patients have access to treatment.