Latest News
Patients With LAL-D May Be at an Increased Risk of Hepatocellular Carcinoma
New Assay Can Identify Patients With Lysosomal Acid Lipase Deficiency
NGS Detects Structural Variants in Diseases Including LAL-D and Pompe Disease
Infantile Hepatosplenomegaly in LAL-D: A Case Report
Rare Disease Patients and Advocate to Headline NORD Conference in October
New Next-Generation Sequencing Test Launched for Lipid Disorders Such as LAL-D
Single-Dose Gene Therapy Could Be Potentially Curative for LAL-D
The Feasibility of Screening for LAL-D in At-Risk Populations
Universal Familial Hypercholesterolemia Screening Effective for LAL-D
Latest Clinical Trials
Insights
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.
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.
LAL-D Features
The Alliance for Regenerative Medicine Fights for Better Cell, Gene Therapies
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
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.
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.
Rare Care Podcast
An Update on LAL-D With Dr. Paul Martin of Nationwide Children’s Hospital
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