Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

March 12, 2020March 12, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

January 24, 2020January 24, 2020

Entrada Developing Enzyme Replacement Therapy ENTR-501 to Treat MNGIE Disease

News

Entrada Therapeutics is developing an enzyme replacement therapy, called ENTR-501, as a potential new treatment strategy for people with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). MNGIE is a rare inherited disease caused ... Read more

January 3, 2020January 3, 2020

New Fluorescent Marker Allows Detailed Study of Mitochondria in Live Cells, Study Reports

News

MitoPB Yellow, a new fluorescent specific marker, allows scientists to study live mitochondria, possibly leading to a better understanding of mitochondrial diseases and to new treatments, researchers in Japan ... Read more

November 8, 2019November 8, 2019

Elamipretide Improves Cardiac Function in Barth Syndrome Patients, Phase 2/3 Data Show

News

Elamipretide (MT-131), developed by Stealth BioTherapeutics, is able to improve cardiac function in Barth syndrome patients, new data from a Phase 2/3 clinical trial show. Hilary Vernon, MD, PhD, ... Read more

April 26, 2019April 26, 2019

GS010 Gene Therapy Continues to Improve Vision Clarity in LHON Patients, New Phase 3 Data Show

News

GS010, a gene therapy designed to improve vision clarity in people with Leber hereditary optic neuropathy (LHON), continues to show benefits in the second scheduled readout — at week 72 — ... Read more

March 29, 2019March 29, 2019

New Phase 1 Trial of Potential Treatment for Mitochondrial Diseases, KL1333, Enrolling in UK

News

A second early clinical trial evaluating KL1333, an investigational treatment for genetic mitochondrial diseases, has enrolled its first participant, NeuroVive announced. The Phase 1a/b trial (NCT03888716) of this small molecule treatment ... Read more

October 19, 2018October 19, 2018

NV354, Potential Therapy for Mitochondrial Diseases, Shows Promise in Preclinical Studies

News

NeuroVive Pharmaceutical’s therapeutic candidate NV354 for mitochondrial diseases, such as Leigh syndrome, was able to restore the body’s own energy substrate — succinate — in an in vivo model of ... Read more

August 24, 2018August 24, 2018

Short-Term Treatment With KH176 Lessens Depression, Improves Mood in Mitochondria Patients, Study Shows

News

Short-term treatment with oral KH176 was safe and well-tolerated by mitochondria disease patients, and lessened their symptoms of depression while also having a positive effect in alertness and mood, ... Read more

July 20, 2018July 20, 2018

HotSpot Therapeutics Secures $45M to Advance Drug Discovery Platform

News

HotSpot Therapeutics has secured $45 million to advance its drug discovery strategy that identifies so-called “hot spots” in proteins, allowing for their precise targeted regulation. Using this technology, HotSpot Therapeutics ... Read more

June 15, 2018June 15, 2018

Mutations in Mitochondrial Gene ANT1 Linked to Risk of Bipolar Disease in Mouse Study

News

Mutations in the mitochondrial gene ANT1 may increase the risk of bipolar disease by altering levels of serotonin transmission in the brain, a mouse study suggests. The study, “Ant1 mutant mice ... Read more