Treating Lysosomal Storage Diseases: More Options at Hand but Challenges Remain
Enzyme therapy for lysosomal storage diseases has evolved recently; however, current enzyme therapies have limitations.
LAL-D Insights
The Challenges of Detecting Lysosomal Acid Lipase Deficiency
Lysosomal acid lipase deficiency is such a rare disease that even screening among at-risk populations may yield no results.
Drug Repurposing: A Novel Strategy for a Deeply Unequal World
The belief driving drug repurposing is that the current drugs that we do have may yet possess therapeutic aspects we aren’t fully aware of.
Supporting Patients and Their Parents Upended by an Unexpected Diagnosis
It is vital that physicians support children diagnosed with lysosomal acid lipase deficiency, as well as their parents/caregivers.
ANCA Insights, HAE Insights, Insights, LAL-D Insights, LCFAOD Insights, MG Insights, MTC Insights, NMOSD Insights, Pompe Insights, SM Insights, WD Insights
Exploring the Parental Burden of Care of Children With Rare Diseases
The parental burden of care of children with rare diseases is considerable and deserves due attention and support.
Liver Disease and Atherosclerosis as Signs of Lysosomal Acid Lipase Deficiency
Studies indicate that concurrent liver disease and atherosclerosis can sometimes be signs of a lysosomal storage disorder.
Investigating the Merits of an LAL-D Screening Program
Lysosomal acid lipase deficiency screening programs can identify cases early, but is it worth the investment?
Improving the Detection and Diagnosis of Lysosomal Acid Lipase Deficiency
Physicians should suspect a diagnosis of LAL-D if the patient has a relevant clinical presentation and family history.
Evolving Perspectives on the Role of Lysosomes in Maintaining Cellular Homeostasis
Lysosomal storage diseases, including lysosomal acid lipase deficiency, were first described in the 19th century, even before lysosomes were clearly defined in biology. They were initially characterized as a group of symptoms—as opposed to the molecular diagnosis we use today. “Knowledge on lysosomal storage diseases has been evolving for more than a century,” Parenti and…
Understanding Bone Disease Associated With Lysosomal Acid Lipase Deficiency
Bone pathology can occur as a result of lysosomal storage diseases such as lysosomal acid lipase deficiency and Gaucher disease.
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