Posts by: Iqra Mumal

News

Entrada Therapeutics has raised $59 million in Series A financing to advance its intracellular enzyme replacement therapy program for treatment of a rare and deadly mitochondrial disease. Series A financing ... Read more

September 20, 2018September 20, 2018

PISD is a Novel Mitochondrial Disease Gene, Case Report Shows

News

Mutations in the PISD gene can cause mitochondrial disease, a new study shows. The report, “PISD is a mitochondrial disease gene causing skeletal dysplasia, cataracts and white matter changes,” appeared in ... Read more

September 14, 2018September 14, 2018

Elesclomol Can Help Restore Copper Levels in Patients with Mitochondrial Diseases, Researchers Find

News

Elesclomol, an investigational anti-cancer drug, can help treat patients with mitochondrial diseases by restoring copper in the mitochondria, a new study shows. The study, “Elesclomol restores mitochondrial function in ... Read more

June 29, 2018June 29, 2018

Older Moms More Likely to Pass Along Mitochondrial DNA with Mutations, Study Finds

News

A mathematical model that can predict the nature of the mitochondrial DNA a child will inherit from its mother was part of a study that also found that maternal ... Read more

June 1, 2018June 1, 2018

Surveys Details Reasons Mitochondrial Disease Patients Take Part – or Don’t – in Clinical Trials

News

Two surveys of people with mitochondrial disease spotlighted factors that both motivate and hinder patients from taking part in clinical trials, and underscored the importance of working with patient ... Read more

May 25, 2018May 25, 2018

Phase 1 Clinical Trial Testing Potential Mitochondrial Disease Therapy KL1333 Shows Positive Results

News

NeuroVive Pharmaceutical AB and Yungjin Pharm announced positive topline results from a Phase 1 clinical trial evaluating KL1333, an investigational treatment for genetic mitochondrial diseases. The randomized, single ascending dose trial ... Read more

March 16, 2018March 16, 2018

Protein That Helps Our Circadian Clocks and Mitochondria Work Together Identified in Study

News

Exactly how the circadian rhythm — our 24-hour internal clocks — and mitochrondria — our cell’s energy producers — cross talk to coordinate their work and maintain cellular health appears ... Read more

March 14, 2018March 14, 2018

Scientists May Have Found New Mechanism Behind Mitochondrial Disease Development

News

Errors in the chemical processing that takes place in the production of mitochondrial proteins may play a role in the development of certain mitochondrial diseases, scientists from Japan have ... Read more

February 16, 2018

Heart, Brain Very Sensitive to Mitochondrial Defects in Early Development, Study Shows

News

Mutations in genes involved in the production of mitochondrial proteins may affect the heart and brain severely during early development and lead to a poor prognosis, researchers have found. ... Read more

February 14, 2018

Researchers Identify New Gene Variant Related to Childhood Mitochondrial Disease

News

Researchers used next-generation sequencing to identify NDUFB8 as a relevant gene in childhood-onset mitochondrial disease, a new study shows. The study, “NDUFB8 Mutations Cause Mitochondrial Complex I Deficiency in Individuals with Leigh-like ... Read more