The CLN3 gene may play a more crucial role than previously thought in the normal balance of cellular recycling…
Articles by Iqra Mumal, PhD
Exome sequencing, a technique that looks for gene mutations, is a valuable tool to diagnose pediatric neuronal ceroid lipofuscinoses, also…
The U.S. Food and Drug Administration has accepted a new drug application (NDA) for UCB’s Nayzilam (midazolam) nasal…
Substantial progress has been made in the development of gene therapies for Batten disease, according to a recent review, and…
A family of enzymes called metallothioneins potentially could be new therapeutic agents in the treatment of lysosomal storage disorders (LSDs),…
A recent review highlights how autophagy, a process in the body that deals with the destruction of cells to…
An analysis of the protein profile of the three major forms of neuronal ceroid lipofuscinoses (NCLs) has revealed potential biomarkers…
New research shows that patients with infantile Batten disease exhibit significant early disease in the spinal cord and that combined…
The fat-lowering therapy gemfribrozil decreased disability and increased the lifespan of mice with a form of Batten disease that strikes children,…
Compounds that inhibit the effects of enzymes linked to brain inflammation improve movement in mice with both early and advanced…