Latest News
Rapid Whole Genome Sequencing Facilitates Early Treatment of LAL-D
People With Rare Diseases Report Facing Stigma, Poor Quality of Life
Clinicians: Long-Term ERT Leads to Variable Improvements in LAL-D
Could Mass Spectrometry or Fluorimetry Be Used in Diagnosing Pompe Disease?
New Newborn Screening Program for LAL-D Available in New York
LAL-D Leads to Feelings of Uncertainty and Powerlessness in Parents
Study Suggests Reinvestigating Inhibitor Concentrations in LAL-D
Sebelipase Alfa Provides Sustained Improvements in LAL-D Trial
Hepatocyte-Like Cells in 3D Differentiation Useful for LAL-D Therapy
Latest Clinical Trials
Features
Overcoming the Clinical Challenge of Detecting and Diagnosing LAL-D
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.
Rare Disease Day 2022: Health Care Policy and Protections Still Lacking in Many States
NORD and other rare disease organizations around the world will celebrate Rare Disease Day on February 28, 2022, with most events being held virtually.
Using Technology to Boost Ethnic, Geographic Diversity in Clinical Trials
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.
CORD Aims to Improve Diagnosis, Drug Access for Canada’s Rare Disease Patients
The Canadian Organization for Rare Disorders advocates for better diagnosis and drug access for Canada’s 3 million people with rare diseases.
Gene Therapy May Offer Hope for Patients With LAL-D, a Disease With No Known Cure
Researchers are investigating gene therapy for use in the treatment of lysosomal acid lipase deficiency (LAL-D).
LAL-D Clinical Insights
The Relationship Between Inborn Errors of Metabolism and Pediatric Dental Disorders
We shine a spotlight on a lesser-known implication of inborn errors of metabolism: dental effects that require treatment by a dentist.
Is Large-Scale Screening Suitable for Detecting Lysosomal Acid Lipase Deficiency?
We examine 2 studies that evaluate the suitability of large-scale screening for lysosomal acid lipase deficiency.
The Role of Lentiviral Vectors in Treating Lysosomal Storage Diseases
Lentiviral vectors provide hope for a cure in a number of lysosomal storage diseases and other disorders and malignancies.
The Far-Reaching Impact of Innovative Clinical Trial Designs in Rare Disease
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
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