We shine a spotlight on a lesser-known implication of inborn errors of metabolism: dental effects that require treatment by a dentist.
We examine 2 studies that evaluate the suitability of large-scale screening for lysosomal acid lipase deficiency.
Lentiviral vectors provide hope for a cure in a number of lysosomal storage diseases and other disorders and malignancies.
We examine the lasting impact of innovative clinical trial designs for rare diseases on other clinical experiments.
Lysosomal acid lipase deficiency (LAL-D) is one of many possible genetic causes of adrenal insufficiency.
We explore the rarely discussed relationship between lysosomal acid lipase deficiency (LAL-D) and bone homeostasis.
We explore how physicians can recognize some of the first signs of LAL-D to have a better chance of diagnosing the disease early.
We take a close look at the hepatic manifestations of lysosomal acid lipase deficiency (LAL-D), as well as currently available diagnostic tools.
Treatment of lysosomal acid lipase deficiency, which presents with different phenotype severity, is still challenging despite recent advances.