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Ataxia is a neurological sign and symptom that consists of gross lack of coordination of muscle movements. Ataxia is a non-specific clinical manifestation implying disfunction of the parts of the nervous system that coordinate movement, such as the cerebellum.
FDA accepts Eisai's sNDA for perampanel drug for treatment of PGTC seizures

FDA accepts Eisai's sNDA for perampanel drug for treatment of PGTC seizures

Eisai Inc. announced today that the U.S. Food and Drug Administration (FDA) accepted for review the company's Supplemental New Drug Application (sNDA) for its in-house-discovered AMPA receptor antagonist perampanel for the treatment of primary generalized tonic-clonic (PGTC) seizures. [More]
Study shows inactivation of p53 protein promotes cancer progression

Study shows inactivation of p53 protein promotes cancer progression

Researchers at the Bellvitge Biomedical Research Institute (IDIBELL) and the University of Barcelona have discovered the interaction between HERC2 proteins with another protein called p53 that is inactivated in more than half of human tumors. [More]
Edison's EPI-743 receives FDA orphan drug designation for treatment of Leigh syndrome

Edison's EPI-743 receives FDA orphan drug designation for treatment of Leigh syndrome

Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome. [More]
New research shows how misfolded proteins are precisely selected for degradation

New research shows how misfolded proteins are precisely selected for degradation

It's almost axiomatic that misfolded proteins compromise how cells normally function and cause debilitating human disease, but how these proteins are detected and degraded within the body is not well understood. [More]
Eisai launches new class of epilepsy treatment Fycompa in France

Eisai launches new class of epilepsy treatment Fycompa in France

Fycompa (perampanel), the first in an entirely new class of treatment for partial onset seizures (the most common form of epilepsy), has today received reimbursement approval from the Comité Economique des Produits de Santé. [More]
Scientists develop new way to decode specific aspects of movement in human brain

Scientists develop new way to decode specific aspects of movement in human brain

Our human brains are filled with maps: visual maps of our external environments, and motor maps that define how we interact physically within those environments. Somehow these separate points of reference need to correspond with - and to - one another in order for us to act, whether it's grasping a coffee cup or hitting a tennis ball. [More]
Study of rare childhood neurodegenerative diseases identifies new source of DNA damage

Study of rare childhood neurodegenerative diseases identifies new source of DNA damage

St. Jude Children's Research Hospital scientists studying two rare, inherited childhood neurodegenerative disorders have identified a new, possibly common source of DNA damage that may play a role in other neurodegenerative diseases, cancer and aging. [More]
Diagnosis of Huntington's disease requires combination of clinical symptoms, radiological changes, genetic diagnosis

Diagnosis of Huntington's disease requires combination of clinical symptoms, radiological changes, genetic diagnosis

Huntington's disease is an autosomal-dominant inherited neurodegenerative disease with a distinct phenotype, but the pathogenesis is unclear. [More]
Research suggests that person can slow aging process by exercising regularly

Research suggests that person can slow aging process by exercising regularly

New research by Canadian sports medicine physician Mark Tarnopolsky, MD, PhD, suggests that a person can slow the speed at which they age by exercising regularly. Dr. Tarnopolsky presented his research titled, "Exercise as a Countermeasure for Aging: From Mice to Humans" today at the 23rd Annual Meeting of the American Medical Society for Sports Medicine (AMSSM). Dr. Tarnopolsky discussed how regular exercise not only improves the quality of life but can also extend a person's lifespan by up to five years. [More]
Expert in gene therapy joins The Children's Hospital of Philadelphia

Expert in gene therapy joins The Children's Hospital of Philadelphia

Beverly L. Davidson, Ph.D., a nationally prominent expert in gene therapy, is joining The Children's Hospital of Philadelphia (CHOP) today. [More]
Scientists discover why cerebellar granule cell neurons in A-T patients fail to repair DNA damage

Scientists discover why cerebellar granule cell neurons in A-T patients fail to repair DNA damage

A team of scientists, led by Stuart Lipton, M.D., Ph.D., professor and director of the Neuroscience and Aging Research Center at Sanford-Burnham Medical Research Institute, recently discovered why cerebellar granule cell neurons in patients suffering from ataxia-telangiectasia (A-T) were unable to repair DNA damage and thus died. [More]
Edison Pharmaceuticals begins EPI-743 clinical trial in children with Pearson syndrome

Edison Pharmaceuticals begins EPI-743 clinical trial in children with Pearson syndrome

Edison Pharmaceuticals today announced the initiation of a phase 2 study entitled "A Phase 2 Safety and Efficacy Study of EPI-743 (Vincerinone™) in Children with Pearson Syndrome." The Investigative New Drug application (IND) was approved by the Food and Drug Administration, Office of Hematology and Oncology Products. [More]
FDA grants Fast Track designation to Edison’s lead drug EPI-743

FDA grants Fast Track designation to Edison’s lead drug EPI-743

Edison Pharmaceuticals today announced that the United States Food and Drug Administration granted Fast Track designation to EPI-743, the company's lead drug, for the treatment of Friedreich's ataxia. EPI-743 is being developed for pediatric and adult mitochondrial disease, including Friedreich's ataxia. [More]
Edison Pharmaceuticals initiates Phase 2B/3 study of EPI-743 in children with Leigh syndrome

Edison Pharmaceuticals initiates Phase 2B/3 study of EPI-743 in children with Leigh syndrome

Edison Pharmaceuticals today announced the initiation of a phase 2B/3 study entitled, "A Phase 2B/3 Open-label Study of EPI-743 in Children with Leigh Syndrome" to be conducted in conjunction with Dainippon Sumitomo Pharma Co, Ltd. [More]
Longtime allies team up to create Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence

Longtime allies team up to create Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence

Three longtime allies have joined forces to create the new Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence. [More]
UMMS launches Voyager Therapeutics to develop life-changing treatments for fatal diseases

UMMS launches Voyager Therapeutics to develop life-changing treatments for fatal diseases

UMass Medical School (UMMS) recognizes the launch today of Voyager Therapeutics, a gene therapy company founded by four world leaders in the fields of AAV gene therapy, RNA biology and neuroscience, to develop life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS). [More]
Researchers find link between mutations in gene called RAB 24 and inherited neurodegenerative disease

Researchers find link between mutations in gene called RAB 24 and inherited neurodegenerative disease

Researchers from North Carolina State University have found a link between a mutation in a gene called RAB 24 and an inherited neurodegenerative disease in Old English sheepdogs and Gordon setters. The findings may help further understanding of neurodegenerative diseases and identify new treatments for both canine and human sufferers. [More]
Edison's vatiquinone receives Orphan Status for treatment of Friedreich's ataxia

Edison's vatiquinone receives Orphan Status for treatment of Friedreich's ataxia

Edison Pharmaceuticals today announced that the US Food and Drug Administration has granted Orphan Status to vatiquinone for the treatment of Friedreich's ataxia. [More]
FARA funds gene therapy-based research project to tackle Friedreich's ataxia

FARA funds gene therapy-based research project to tackle Friedreich's ataxia

The gene therapy-based research project to tackle Friedreich's ataxia launched in November in labs at the Institute for Research in Biomedicine, in Barcelona, and the "Centro de Biolog-a Molecular Severo Ochoa", in Madrid, has received 100,000 US$ per year for two years from the Friedreich's Ataxia Research Alliance. [More]
Merck and CNIO sign agreement to collaborate in area of cancer drug development

Merck and CNIO sign agreement to collaborate in area of cancer drug development

The Spanish National Cancer Research Centre (CNIO) and the German chemical and pharmaceutical company Merck today signed an agreement in Madrid to collaborate in the area of cancer drug development. [More]