Posts by: Iqra Mumal, PhD

News

The CLN3 gene may play a more crucial role than previously thought in the normal balance of cellular recycling processes and the degradation of important fatty molecules in nervous ... Read more

September 25, 2018September 25, 2018

Exome Sequencing is a Valuable Tool for Diagnosis of Pediatric Batten Disease

News

Exome sequencing, a technique that looks for gene mutations, is a valuable tool to diagnose pediatric neuronal ceroid lipofuscinoses, also known as Batten disease, according to a new case ... Read more

August 22, 2018August 22, 2018

FDA Accepts New Drug Application for Nayzilam Nasal Spray for Acute Treatment of Seizures

News

The U.S. Food and Drug Administration has accepted a new drug application (NDA) for UCB’s Nayzilam (midazolam) nasal spray for the acute treatment of seizures including seizure clusters and acute ... Read more

May 11, 2018May 11, 2018

Study Examines Promising Advances in Gene Therapies to Treat Batten Disease

News

Substantial progress has been made in the development of gene therapies for Batten disease, according to a recent review, and researchers predict that targeted combination therapies will improve patients’ ... Read more

February 23, 2018March 23, 2018

Metallothioneins Show Promise as Therapeutic Targets in Mouse Model of Batten Disease

News

A family of enzymes called metallothioneins potentially could be new therapeutic agents in the treatment of lysosomal storage disorders (LSDs), including Batten disease, a mouse model shows. The study, ... Read more

February 6, 2018February 6, 2018

Abnormal Autophagy Is Common in Lysosomal Storage Diseases, Including Batten

News

A recent review highlights how autophagy, a process in the body that deals with the destruction of cells to maintain balance and normal functions, is impaired in patients with ... Read more

August 24, 2017

Potential Batten Biomarkers Identified Through Protein Analyses

News

An analysis of the protein profile of the three major forms of neuronal ceroid lipofuscinoses (NCLs) has revealed potential biomarkers for the measurement of short-term response to treatment. The ... Read more

July 13, 2017

Infantile Batten Disease May Begin in Spinal Cord Rather Than Brain, Study Suggests

News

New research shows that patients with infantile Batten disease exhibit significant early disease in the spinal cord and that combined treatment of the spinal cord and the brain has ... Read more

June 21, 2017

Cholesterol Therapy Gemfibrozil Improves Disability and Lifespan of Mice with Batten Disease, Study Reports

News

The fat-lowering therapy gemfribrozil decreased disability and increased the lifespan of mice with a form of Batten disease that strikes children, according to a preclinical-trial study. The finding suggests the ... Read more

May 16, 2017

PDE4 Enzyme Inhibitors Improve Movement in Mice with Batten Disease, Study Shows

News

Compounds that inhibit the effects of enzymes linked to brain inflammation improve movement in mice with both early and advanced Batten disease, according to a study. The compounds are ... Read more

Posts by: Iqra Mumal, PhD

Iqra Mumal, PhD

CLN3 Deficiency Leads to Abnormalities in Cellular Recycling Process, Study Shows

Exome Sequencing is a Valuable Tool for Diagnosis of Pediatric Batten Disease

FDA Accepts New Drug Application for Nayzilam Nasal Spray for Acute Treatment of Seizures

Study Examines Promising Advances in Gene Therapies to Treat Batten Disease

Metallothioneins Show Promise as Therapeutic Targets in Mouse Model of Batten Disease

Abnormal Autophagy Is Common in Lysosomal Storage Diseases, Including Batten

Potential Batten Biomarkers Identified Through Protein Analyses

Infantile Batten Disease May Begin in Spinal Cord Rather Than Brain, Study Suggests

Cholesterol Therapy Gemfibrozil Improves Disability and Lifespan of Mice with Batten Disease, Study Reports

PDE4 Enzyme Inhibitors Improve Movement in Mice with Batten Disease, Study Shows

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