Latest Clinical Trials
Bone pathology can occur as a result of lysosomal storage diseases such as lysosomal acid lipase deficiency and Gaucher disease.
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.
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
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.
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.
Rare Care Podcast
An Update on LAL-D With Dr. Paul Martin of Nationwide Children’s Hospital