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.
FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration has cleared the Company's Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type I (MPS I). [More]
Lamin nucleoskeleton disordered in Alzheimer's

Lamin nucleoskeleton disordered in Alzheimer's

Brain cell death in Alzheimer's disease is linked to disruption of a skeleton that surrounds the nucleus of the cells, a researcher in the School of Medicine at The University of Texas Health Science Center at San Antonio said. [More]
Study finding could shed light on molecular mechanisms underlying Huntington's disease

Study finding could shed light on molecular mechanisms underlying Huntington's disease

Researchers at the University of Pittsburgh School of Medicine have shown that the core of the protein clumps found in the brains of people with Huntington's disease have a distinctive structure, a finding that could shed light on the molecular mechanisms underlying the neurodegenerative disorder. [More]
FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

Ionis Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to IONIS-HTTRx for the treatment of patients with Huntington's disease (HD). [More]
Sangamo presents Phase 2 data from two ongoing clinical trials of SB-728-T for treatment of HIV/AIDS

Sangamo presents Phase 2 data from two ongoing clinical trials of SB-728-T for treatment of HIV/AIDS

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced the presentation of Phase 2 data from two of the Company's ongoing clinical trials (SB-728-1101 Cohort 3* and SB-728-mR-1401) of SB-728-T, which is being developed for the functional control of HIV/AIDS. [More]
Tetra begins human Phase 1 safety trials of BPN14770

Tetra begins human Phase 1 safety trials of BPN14770

Tetra Discovery Partners today announced that the company has initiated human Phase 1 safety trials of its lead compound BPN14770, which Tetra is developing as a potential treatment to both improve memory and slow the progression of Alzheimer's disease. [More]
Existing compound offers hope for slowing Huntington's disease progression

Existing compound offers hope for slowing Huntington's disease progression

Currently, there is no treatment to halt the progression of Huntington's disease (HD), a fatal genetic disorder that slowly robs sufferers of their physical and mental abilities. Now, researchers at University of California, San Diego School of Medicine have discovered that an existing compound, previously tested for diabetes, offers hope for slowing HD and its symptoms. [More]
UF Health investigators make new discovery about Huntington's disease

UF Health investigators make new discovery about Huntington's disease

University of Florida Health researchers have made a new discovery about Huntington's disease, showing that the gene that causes the fatal disorder makes an unexpected "cocktail" of mutant proteins that accumulate in the brain. [More]
Utah chemists devise new way to detect DNA damage

Utah chemists devise new way to detect DNA damage

University of Utah chemists devised a new way to detect chemical damage to DNA that sometimes leads to genetic mutations responsible for many diseases, including various cancers and neurological disorders. [More]
Scientists identify new jelly-like properties of essential proteins that lead to serious diseases

Scientists identify new jelly-like properties of essential proteins that lead to serious diseases

Scientists at the University of Cambridge have identified a new property of essential proteins which, when it malfunctions, can cause the build up, or 'aggregation', of misshaped proteins and lead to serious diseases. [More]
Study reveals mechanism behind protein-related diseases

Study reveals mechanism behind protein-related diseases

Dartmouth researchers have found that some proteins turn into liquid droplets on the way to becoming toxic solids implicated in neurodegenerative diseases and other genetic disorders. [More]
Psychiatric symptoms predate motor onset in Huntington’s disease

Psychiatric symptoms predate motor onset in Huntington’s disease

Prodromal psychiatric symptoms may be more common in patients with Huntington’s disease than previously thought and worsen as the condition progresses, longitudinal study findings show. [More]
First patients receive doses of antisense drug for Huntington's disease

First patients receive doses of antisense drug for Huntington's disease

The first few patients have received doses of an experimental RNA-targeting drug for Huntington's disease, it was announced today. [More]
UB study sheds light on the root causes of Huntington's disease

UB study sheds light on the root causes of Huntington's disease

We've known for years that the Huntingtin protein (Htt) is responsible for Huntington's disease, a neurodegenerative disorder that diminishes a person's mental and physical abilities. [More]
Omeros launches OMIDRIAssure program to provide expanded patient access to Omidria

Omeros launches OMIDRIAssure program to provide expanded patient access to Omidria

Omeros Corporation today announced that it has launched OMIDRIAssure, a comprehensive reimbursement services program to provide expanded access to Omidria® (phenylephrine and ketorolac injection) 1% / 0.3% for cataract surgery patients and reimbursement assistance for physicians and facilities. [More]
Developing tailored interventions for cognitive and behavioral symptoms key to improving outcomes after TBI

Developing tailored interventions for cognitive and behavioral symptoms key to improving outcomes after TBI

Kessler researchers assessed moderate to severe traumatic brain injury (TBI) for frontal behavioral syndromes and cognitive function. Their finding suggest that adding the Frontal Systems Behavior Scale to neuropsychological assessment may yield useful information about the behavioral sequelae of TBI. [More]
UTHealth biochemists expose structures of IP3R calcium channels

UTHealth biochemists expose structures of IP3R calcium channels

Calcium is a crucial element in the body that controls thought, movement and other bodily functions. These events are directed by specialized proteins called ion channels that allow the flow of calcium ions in and out of cells and among cell compartments. For years, scientists have been unsure how calcium ion channels function. [More]
Lonza, Benitec partner to develop scalable manufacturing process for AAV-based viral gene therapies

Lonza, Benitec partner to develop scalable manufacturing process for AAV-based viral gene therapies

Lonza Houston, Inc., a global leader in biological, cell and viral gene therapy manufacturing, and Benitec Biopharma, a clinical stage biotechnology company commercializing a patented gene-silencing technology, DNA-directed RNA interference (ddRNAi), today announced that they have entered into a Manufacturing Services Agreement to develop a scalable manufacturing process for Benitec's ddRNAi-based, Adeno-Associated Virus (AAV)-delivered products intended for therapeutic use in humans. [More]
Songbirds may help researchers find cure for Huntington’s disease

Songbirds may help researchers find cure for Huntington’s disease

Many neurological disorders can rob someone of the ability to speak clearly, causing them to stutter, mispronounce words, and struggle to put together coherent sentences. However, the molecular and neurological dysfunctions that cause these symptoms aren't well understood. Recent work at The Rockefeller University may give researchers a new tool to better study these vocal and speech impairments, particularly in Huntington's disease. [More]
Novel approach promises easier way to detect protein fibers implicated in neuro-degenerative diseases

Novel approach promises easier way to detect protein fibers implicated in neuro-degenerative diseases

Liquid crystals are familiar to most of us as the somewhat humdrum stuff used to make computer displays and TVs. Even for scientists, it has not been easy to find other ways of using them. [More]
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