Latest Clinical Trials
Lysosomal acid lipase deficiency is so rare that most doctors have never heard of it, let alone treated a patient with the disease, says Henry Lin, MD, MBA.
NORD and other rare disease organizations around the world will celebrate Rare Disease Day on February 28, 2022, with most events being held virtually.
Eliminating geographic and ethnic bias in clinical trials for rare diseases is the goal of Jeeva Informatics, says founder and CEO Harsha K. Rajasimha, PhD.
The Canadian Organization for Rare Disorders advocates for better diagnosis and drug access for Canada’s 3 million people with rare diseases.
Researchers are investigating gene therapy for use in the treatment of lysosomal acid lipase deficiency (LAL-D).
LAL-D Clinical Insights
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.
Rare Care Podcast
An Update on LAL-D With Dr. Paul Martin of Nationwide Children’s Hospital