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Auxilium Pharmaceuticals' CCH drug effective against cellulite in Phase 2a study

Auxilium Pharmaceuticals' CCH drug effective against cellulite in Phase 2a study

Auxilium Pharmaceuticals, Inc., a specialty biopharmaceutical company, today announced positive results from a randomized, double-blind Phase 2a study of collagenase clostridium histolyticum (or CCH) for the treatment of edematous fibrosclerotic panniculopathy (EFP), commonly known as cellulite. [More]
New articles shed light on PTSD, other mental health problems

New articles shed light on PTSD, other mental health problems

Published today in the open access European Journal of Psychotraumatology is a collection of articles that offer rare and unique insight into the often fiercely debated topic of deployment related PTSD and associated mental health problems. [More]
Study identifies novel gene that controls nerve conduction velocity linked with MS

Study identifies novel gene that controls nerve conduction velocity linked with MS

A new study published in The American Journal of Pathology identifies a novel gene that controls nerve conduction velocity. [More]
Schizophrenia-linked genetic variations and the developing brain: an interview with Prof. Guo-li Ming

Schizophrenia-linked genetic variations and the developing brain: an interview with Prof. Guo-li Ming

How much is currently known about what happens in the developing brain that puts people at risk of schizophrenia? [More]
European Commission approves EYLEA Injection for treatment of visual impairment due to DME

European Commission approves EYLEA Injection for treatment of visual impairment due to DME

Regeneron Pharmaceuticals, Inc. today announced that EYLEA® (aflibercept) Injection has been approved by the European Commission for the treatment of visual impairment due to Diabetic Macular Edema. [More]
FDA approves EYLEA Injection for treatment of Diabetic Macular Edema

FDA approves EYLEA Injection for treatment of Diabetic Macular Edema

Regeneron Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration has approved EYLEA (aflibercept) Injection for the treatment of Diabetic Macular Edema (DME). [More]
Study reveals novel epilepsy pathway linked to neurodegenerative diseases

Study reveals novel epilepsy pathway linked to neurodegenerative diseases

A recent scientific discovery showed that mutations in prickle genes cause epilepsy, which in humans is a brain disorder characterized by repeated seizures over time. However, the mechanism responsible for generating prickle-associated seizures was unknown. [More]
Researchers collaborate to tackle rare diseases

Researchers collaborate to tackle rare diseases

Support from a network of leading researchers across Europe specialised in a rare auto-immune disease with unmet medical needs could help test several novel treatments [More]
Researchers propose ‘microbiome cloud model’ to understand variation in individual's microbiome composition

Researchers propose ‘microbiome cloud model’ to understand variation in individual's microbiome composition

The Human Microbiome Project (HMP) is a global initiative to identify and characterize the microorganisms present at multiple sites in the human body. [More]
New book addresses safe and effective exercises for overweight children

New book addresses safe and effective exercises for overweight children

Safe and Effective Exercise for Overweight Youth by Melinda Sothern, PhD, Director of Behavioral & Community Health Sciences and Professor of Research at the LSU Health Sciences Center New Orleans School of Public Health, provides exercise recommendations and guidelines specifically designed for overweight youth. [More]
Study to understand impairments of working memory among patients with schizophrenia

Study to understand impairments of working memory among patients with schizophrenia

The inability to ignore irrelevant stimuli underlies the impaired working memory and cognition often experienced by individuals diagnosed with schizophrenia, reports a new study in the current issue of Biological Psychiatry. [More]
VENITI’s IDE receives FDA approval for VENITI VICI Venous Stent System

VENITI’s IDE receives FDA approval for VENITI VICI Venous Stent System

VENITI, Inc., a company dedicated to providing innovative technology for the treatment of venous disease, announced that it has received approval from the United States Food and Drug Administration (FDA) for an Investigational Device Exemption (IDE) to begin the VIRTUS trial of the VENITI VICI Venous Stent System. [More]
Fabry disease awareness: an interview with Dr. Hartmann Wellhoefer, Head of Medical Affairs, Rare Disease, Shire

Fabry disease awareness: an interview with Dr. Hartmann Wellhoefer, Head of Medical Affairs, Rare Disease, Shire

Lysosomes are subcellular organelles that are present in most cells, with the major exception of red blood cells. [More]
Galderma's trifarotene molecule gets FDA Orphan Drug Designation for treatment of congenital ichthyosis

Galderma's trifarotene molecule gets FDA Orphan Drug Designation for treatment of congenital ichthyosis

Galderma announces that the U.S. FDA granted Orphan Drug Designation status for the company's trifarotene molecule for the treatment of congenital ichthyosis. Based on this decision, Galderma plans to implement a clinical development plan, reinforcing its commitment to exploring new treatment options for rare diseases, as well as meeting the needs of all patients with skin diseases over the course of their lives. [More]
Cincinnati Children’s, Alexion Pharmaceuticals establish ‘Rare Disease Innovation Fund’

Cincinnati Children’s, Alexion Pharmaceuticals establish ‘Rare Disease Innovation Fund’

Alexion Pharmaceuticals, Inc. and Cincinnati Children's Hospital Medical Center today announced the establishment of a collaboration and fund for the advancement of research in rare disease. [More]
Alexion announces that FDA grants ODD to Soliris for Myasthenia Gravis treatment

Alexion announces that FDA grants ODD to Soliris for Myasthenia Gravis treatment

Alexion Pharmaceuticals (Nasdaq:ALXN) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Soliris® (eculizumab) for the treatment of patients with Myasthenia Gravis (MG), a rare, debilitating neurologic disorder caused by uncontrolled complement activation. [More]
Amygdala alterations during childhood may influence development of anxiety problems

Amygdala alterations during childhood may influence development of anxiety problems

The amygdala is a key "fear center" in the brain. Alterations in the development of the amygdala during childhood may have an important influence on the development of anxiety problems, reports a new study in the current issue of Biological Psychiatry. [More]
New path may lead to powerful adjunctive novel therapy for PTSD treatment

New path may lead to powerful adjunctive novel therapy for PTSD treatment

There are currently only two FDA-approved medications for the treatment of posttraumatic stress disorder (PTSD) in the United States. Both of these medications are serotonin uptake inhibitors. Despite the availability of these medications, many people diagnosed with PTSD remain symptomatic, highlighting the need for new medications for PTSD treatment. [More]
University of Texas professor to receive Robert K. Chalmers Distinguished Pharmacy Educator Award

University of Texas professor to receive Robert K. Chalmers Distinguished Pharmacy Educator Award

The American Association of Colleges of Pharmacy will honor Robert L. Talbert, Pharm.D., B.S., professor of pharmacotherapy in the College of Pharmacy at The University of Texas at Austin, for his excellence as a teacher, his outstanding achievements as an author and mentor, and his overall impact on pharmacy education and the profession. [More]
Cerenis’ SAMBA and MODE Phase II Studies Meet Primary Endpoints for Treatment of FPHA, HoFH

Cerenis’ SAMBA and MODE Phase II Studies Meet Primary Endpoints for Treatment of FPHA, HoFH

Cerenis Therapeutics, the biopharmaceutical company, today announced that two of its Phase II studies, SAMBA and MODE (Modifying Orphan Disease Evaluation), with CER-001, an engineered human apoA-I-containing pre-β HDL mimetic, met their primary clinical endpoints in patients with Familial Primary Hypoalphalipoproteinemia (FPHA) and Homozygous Familial Hypercholesterolemia (HoFH), respectively. [More]