LAL-D REAL STORIES

Meet Maureen & Lauren

Diagnosed with LAL-D at 8 and 12 years old

Lauren was in the first grade when she became ill with persistent abdominal symptoms. Her parents, Susan and Greg, took her to several specialists and discovered that her liver and spleen were enlarged and her liver enzymes were elevated. Lauren’s lab tests were repeated every few months and remained abnormal, but still, no one could pinpoint exactly what was wrong.

Maureen & Lauren, diagnosed with LAL-D at 8 and 12 years old, with their parents and brother

Eventually, Lauren underwent a liver biopsy, which showed severe scarring of the liver. Doctors began to consider the possibility of a genetic disorder and eventually zeroed in on lysosomal acid lipase deficiency (LAL-D), a genetic and progressive rare metabolic disease associated with multi-organ damage in infant, pediatric, and adult patients and premature death in infants.1,2 All 3 Walsh siblings were tested for LAL-D, and it was revealed that not only Lauren, but also older sister Maureen had the disorder. Younger brother Gregory was found to be a carrier of the disease.

The diagnosis was daunting, but the family quickly learned that they had the chance to enroll their daughters in a clinical trial for an investigational medicine. Both continue to manage their disease with their doctors through regular testing and monitoring for changes in signs and symptoms.

“My hope for their future is the same as any parent of any child,” says Greg. “I know that they can deal with this or anything else and move on and live their lives.”

Today, both sisters are in college—Maureen is studying to become a nurse and Lauren is studying elementary education.

Reference:

  1. Bernstein DL, Hülkova H, Bialer MG, Desnick RJ. Cholesteryl ester storage disease: review of the findings in 135 reported patients with an underdiagnosed disease. J Hepatol. 2013;58(6):1230-1243.
  2. Jones SA, Valayannopoulos V, Schneider E, et al. Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants. Genet Med. 2016 May;18(5):452-8.