Huntington's Disease News and Research RSS Feed - Huntington's Disease News and Research

Huntington's disease (HD) results from genetically programmed degeneration of brain cells, called neurons, in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance. HD is a familial disease, passed from parent to child through a mutation in the normal gene. Each child of an HD parent has a 50-50 chance of inheriting the HD gene. If a child does not inherit the HD gene, he or she will not develop the disease and cannot pass it to subsequent generations. A person who inherits the HD gene will sooner or later develop the disease. Whether one child inherits the gene has no bearing on whether others will or will not inherit the gene. Some early symptoms of HD are mood swings, depression, irritability or trouble driving, learning new things, remembering a fact, or making a decision. As the disease progresses, concentration on intellectual tasks becomes increasingly difficult and the patient may have difficulty feeding himself or herself and swallowing. The rate of disease progression and the age of onset vary from person to person. A genetic test, coupled with a complete medical history and neurological and laboratory tests, helps physicians diagnose HD. Presymptomic testing is available for individuals who are at risk for carrying the HD gene. In 1 to 3 percent of individuals with HD, no family history of HD can be found.
Interleukin receives conditional approval to offer results of PerioPredict genetic risk test

Interleukin receives conditional approval to offer results of PerioPredict genetic risk test

Interleukin Genetics, Inc. today announced it has received conditional approval from the New York State Department of Health to offer, process and report the results of the PerioPredict™ Genetic Risk Test for periodontal disease. [More]
MSU research pushes promising molecule toward clinical trials for treatment of neurological disorders

MSU research pushes promising molecule toward clinical trials for treatment of neurological disorders

The most effective way to tackle debilitating diseases is to punch them at the start and keep them from growing. [More]
New mouse study indicates that mutant protein in muscle cells is responsible for SBMA

New mouse study indicates that mutant protein in muscle cells is responsible for SBMA

Sometimes known as Kennedy's disease, spinal and bulbar muscular atrophy (SBMA) is a rare inherited neuromuscular disorder characterized by slowly progressive muscle weakness and atrophy. [More]
Study evaluates co-occurrence of cancers in patients with central nervous system disorders

Study evaluates co-occurrence of cancers in patients with central nervous system disorders

The study evaluated the co-occurrence of cancers in patients with central nervous system disorders, including Alzheimer's disease, amyotrophic lateral sclerosis, autism spectrum disorders, Down's syndrome, Huntington's disease, multiple sclerosis, Parkinson's disease and schizophrenia. [More]

Thomson Reuters, Children's Tumor Foundation partner to create neurofibromatosis pathway maps

The Intellectual Property & Science business of Thomson Reuters, the world's leading provider of intelligent information for businesses and professionals, today announced an expansion of its partnership with the Children's Tumor Foundation to create unique neurofibromatosis (NF) pathway maps aimed at significantly increasing the understanding of the disease and its underlying mechanisms. [More]
FDA clears Omeros' OMS721 IND for inhibition of complement‑mediated TMAs

FDA clears Omeros' OMS721 IND for inhibition of complement‑mediated TMAs

Omeros Corporation today announced that its Investigational New Drug Application (IND) to evaluate OMS721 for the inhibition of complement‑mediated thrombotic microangiopathies (TMAs) has been cleared by the U.S. Food and Drug Administration. [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]
New gene-editing system holds potential for treating many genetic disorders

New gene-editing system holds potential for treating many genetic disorders

Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation. [More]
Potassium boost improves walking, prolongs survival in mouse model of Huntington's disease

Potassium boost improves walking, prolongs survival in mouse model of Huntington's disease

Tweaking a specific cell type's ability to absorb potassium in the brain improved walking and prolonged survival in a mouse model of Huntington's disease, reports a UCLA study published March 30 in the online edition of Nature Neuroscience. The discovery could point to new drug targets for treating the devastating disease, which strikes one in every 20,000 Americans. [More]

Johns Hopkins neuroscientists identify cause of brain degeneration in Huntington's disease

Working with genetically engineered mice, Johns Hopkins neuroscientists report they have identified what they believe is the cause of the vast disintegration of a part of the brain called the corpus striatum in rodents and people with Huntington's disease: loss of the ability to make the amino acid cysteine. [More]
FDA accepts Lundbeck's carbamazepine NDA for review

FDA accepts Lundbeck's carbamazepine NDA for review

Lundbeck LLC today announced that the U.S. Food and Drug Administration has accepted for review a New Drug Application (NDA) for its investigational therapy intravenous carbamazepine, an intravenous formulation of the anti-epileptic drug (AED) carbamazepine. An action letter is anticipated before the end of 2014. Carbella is the proposed U.S. trade name for intravenous carbamazepine. [More]
Omeros submits investigational new drug to initiate OMS721 Phase 2 clinical program

Omeros submits investigational new drug to initiate OMS721 Phase 2 clinical program

Omeros Corporation today announced that it has submitted to the U.S. Food and Drug Administration (FDA) an investigational new drug (IND) application to initiate the Phase 2 clinical program for OMS721, the company's lead human monoclonal antibody targeting mannan-binding lectin‑associated serine protease-2 (MASP-2), the key regulator of the lectin pathway of the immune system. [More]
Protein interaction network provides invaluable resource to identify targets for Huntington's disease

Protein interaction network provides invaluable resource to identify targets for Huntington's disease

Researchers at the Buck Institute have identified and categorized thousands of protein interactions involving huntingtin, the protein responsible for Huntington's disease (HD). To use an analogy of a human social network, the identified proteins are like "friends" and "friends of friends" of the HD protein. [More]

Study provides novel insight into the impact of genes on Huntington's disease

A study led by researchers at Boston University School of Medicine provides novel insight into the impact that genes may have on Huntington's disease (HD). The study, published online in PLOS Genetics, identified specific small segments of RNA (called micro RNA or miRNA) encoded in DNA in the human genome that are highly expressed in HD. [More]

Researchers develop new compound that slows down progression of Huntington's Disease

Juliet Ross is suffering from Huntington's Disease, a rare neurological disorder that affects one in 10.000 people. Due to a genetic defect, her body produces a toxic protein that damages neurons in her brain. [More]
Cystinosis Research Foundation granted $2.15M in 2013 to researchers investigating cure for cystinosis

Cystinosis Research Foundation granted $2.15M in 2013 to researchers investigating cure for cystinosis

The Cystinosis Research Foundation awarded $2,153,048 in 2013 to researchers investigating better treatments and a cure for cystinosis, a rare metabolic and genetic disease that afflicts about 500 children and young adults in the United States and 2,000 worldwide. [More]

Omeros doses first patient in second Phase 2 clinical trial of OMS824

Omeros Corporation today announced dosing of the first patient in a second Phase 2 clinical trial of OMS824, the company's phosphodiesterase 10 (PDE10) inhibitor being developed for the treatment of schizophrenia, Huntington's disease (HD) and other cognitive disorders. The Phase 2 trial will evaluate the tolerability, safety, pharmacokinetics and performance on a battery of tests in patients with symptomatic HD. [More]
RNA interference approach holds great promise for treating cancer and other diseases

RNA interference approach holds great promise for treating cancer and other diseases

Inspired by tiny particles that carry cholesterol through the body, MIT chemical engineers have designed nanoparticles that can deliver snippets of genetic material that turn off disease-causing genes. [More]
New approach for reducing levels of toxic protein fragments associated with Huntington's disease

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

An innovative therapeutic strategy for reducing the levels of toxic protein fragments associated with Huntington's disease uses a new approach called exon skipping to remove the disease-causing component of the essential protein, huntingtin. [More]

Omeros announces additional positive data from OMS824 Phase 1 program

Omeros Corporation today announced additional positive data from its Phase 1 program for OMS824, the lead compound in Omeros' phosphodiesterase 10 (PDE10) program. This clinical trial evaluated the extent to which OMS824 binds to PDE10, an enzyme expressed in the region of the brain that has been linked to a wide range of diseases that affect cognition. In the latest cohort enrolled in this trial, OMS824 achieved a high of approximately 70‑percent engagement at PDE10 without evidence of extrapyramidal symptoms (EPS). [More]