Huntington's Disease News and Research

Latest Huntington's Disease News and Research

FDA provides Orphan Drug Designation to Alnylam’s ALN-TTR02

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has provided Orphan Drug Designation to ALN-TTR02 as a therapeutic for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant clinical manifestations of transthyretin (TTR)-mediated amyloidosis (ATTR).

20 Jun 2012

Implanted device delivers genetically engineered neurotrophic factor directly to the brain

Studies suggest that neurotrophic factors, which play a role in the development and survival of neurons, have significant therapeutic and restorative potential for neurologic diseases such as Huntington's disease. However, clinical applications are limited because these proteins cannot easily cross the blood brain barrier, have a short half-life, and cause serious side effects.

19 Jun 2012

New U-M hESC lines now available for federally-funded research

Six new human embryonic stem cell lines derived at the University of Michigan have just been placed on the U.S. National Institutes of Health's registry, making the cells available for federally-funded research.

15 Jun 2012

New analogues effective in inhibiting NOS activity in Parkinson models

University of Granada researchers have tested melatonin analogues in rats as it inhibits the enzyme nitric oxide synthase (NOS), which is involved in the development of conditions as inflammatory bowel disease, septic shock or rheumatoid arthritis, as well as in neurodegenerative conditions as Huntington's disease, Alzheimer's disease, Parkinson's disease and amyotrophic lateral sclerosis.

14 Jun 2012

dSarm/Sarm1 gene promotes axon destruction after injury

Degeneration of the axon and synapse, the slender projection through which neurons transmit electrical impulses to neighboring cells, is a hallmark of some of the most crippling neurodegenerative and brain diseases such as amyotrophic lateral sclerosis (ALS), Huntington's disease and peripheral neuropathy.

8 Jun 2012

MGH, HSG to conduct PBT2 clinical trial in Huntington's disease

The Huntington Study Group (HSG), under the leadership of Ray Dorsey, M.D. with Johns Hopkins Medical and Diana Rosas, M.D. with Massachusetts General Hospital, is conducting a clinical trial in Huntington's disease (HD) throughout the United States and Australia, "A randomized, double-blind, placebo-controlled, study to assess the safety and tolerability, and efficacy of PBT2 in patients with early to mid-stage Huntington's disease" comparing a 100 mg dose or 250 mg dose versus placebo.

8 Jun 2012

New research into cell-damaging effects of Huntington's disease

New research into the cell-damaging effects of Huntington's disease suggests a potentially new approach for identifying possible therapeutic targets for treating the nerve-destroying disorder.

7 Jun 2012

Alnylam announces results from ALN-VSP Phase I study on liver cancers

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced results today from its Phase I extension study with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement.

5 Jun 2012

Top-line results from Alnylam’s ALN-RSV01 Phase IIb trial on RSV infection

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today top-line results from a Phase IIb trial with ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection in lung transplant patients.

31 May 2012

Use of transplanted induced pluripotent stem cells could lead to new treatments for HD

Researchers from South Korea, Sweden, and the United States have collaborated on a project to restore neuron function to parts of the brain damaged by Huntington's disease (HD) by successfully transplanting HD-induced pluripotent stem cells into animal models.

30 May 2012

Brain cells prune circuits in the brain by eating away excess synapses

We’re born with our brains prewired, but as information comes in from our environment, this circuitry is updated. A study from Boston Children’s Hospital provides a new glimpse of how this happens: Brain cells known as microglia, tuned into the crosstalk between neurons, literally engulf unnecessary connections, known as synapses, and prune them away.

24 May 2012

CHDI, Lundbeck enter collaboration to investigate targeted therapy for Huntington's disease

CHDI Foundation, Inc. and Lundbeck today announced a research collaboration to investigate a targeted therapy for Huntington's disease (HD). Currently, no treatment exists to slow or halt the progression of HD, a challenging hereditary neurodegenerative disease characterized by a triad of behavioral, cognitive, and motor symptoms.

21 May 2012

Regenerative medicine researchers explore innovative ways to treat childhood conditions

About 3 percent of the babies born in the United States have a birth defect. Children without a birth defect are also susceptible to injury or disease. At The University of Texas Health Science Center at Houston (UTHealth), regenerative medicine researchers are exploring innovative ways to treat these conditions.

21 May 2012

Sigmoid provides financial and product development update

Sigmoid Pharma Ltd., a privately-owned, specialty pharmaceutical and drug delivery company registered in Ireland, today provides an update on significant progress in 2011 and 2012.

19 May 2012

CHDI, KineMed partner to investigate turnover rates of huntingtin protein

KineMed, Inc. and CHDI Foundation, Inc. announced today a collaboration to use KineMed's isotopic labeling and dynamic proteomics platform to investigate the turnover rates of huntingtin protein, a mutant form of which plays a central role in the pathogenesis of Huntington's disease (HD).

15 May 2012

Final results from Alnylam’s ALN-TTR01 Phase I trial on TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today final results from its completed Phase I clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis.

11 May 2012

Alnylam first quarter revenues decrease to $20.6 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter 2012, and company highlights.

4 May 2012

Couples with infertility and miscarriages offered detailed embryo screening with 99% accuracy

Termed “preimplantation genetic screening (PGS)” the process involves a full chromosome count of embryos to ensure only the healthiest are implanted. It has a 99 per cent accuracy rate, giving hopeful parents the best chance to conceive and carry a healthy baby to term.

30 Apr 2012

Researchers examines whether MRI can improve accuracy of Huntington’s diagnosis

Huntington disease (HD) is an inherited neurodegenerative disorder caused by a defect on chromosome four where, within the Huntingtin gene, a CAG repeat occurs too many times. Most individuals begin experiencing symptoms in their 40s or 50s, but studies have shown that significant brain atrophy occurs several years prior to an official HD diagnosis. As a result, the field has sought a preventive treatment that could be administered prior to the development of actual symptoms that might delay the onset of illness.

27 Apr 2012

Researchers identify gene that increases risk of MI in newborns with CF

Researchers at Johns Hopkins have identified a gene that modifies the risk of newborns with cystic fibrosis (CF) developing neonatal intestinal obstruction, a potentially lethal complication of CF.

24 Apr 2012

Huntington's Disease News and Research

Latest Huntington's Disease News and Research

FDA provides Orphan Drug Designation to Alnylam’s ALN-TTR02

Implanted device delivers genetically engineered neurotrophic factor directly to the brain

New U-M hESC lines now available for federally-funded research

New analogues effective in inhibiting NOS activity in Parkinson models

dSarm/Sarm1 gene promotes axon destruction after injury

MGH, HSG to conduct PBT2 clinical trial in Huntington's disease

New research into cell-damaging effects of Huntington's disease

Alnylam announces results from ALN-VSP Phase I study on liver cancers

Top-line results from Alnylam’s ALN-RSV01 Phase IIb trial on RSV infection

Use of transplanted induced pluripotent stem cells could lead to new treatments for HD

Brain cells prune circuits in the brain by eating away excess synapses

CHDI, Lundbeck enter collaboration to investigate targeted therapy for Huntington's disease

Regenerative medicine researchers explore innovative ways to treat childhood conditions

Sigmoid provides financial and product development update

CHDI, KineMed partner to investigate turnover rates of huntingtin protein

Final results from Alnylam’s ALN-TTR01 Phase I trial on TTR-mediated amyloidosis

Alnylam first quarter revenues decrease to $20.6 million

Couples with infertility and miscarriages offered detailed embryo screening with 99% accuracy

Researchers examines whether MRI can improve accuracy of Huntington’s diagnosis

Researchers identify gene that increases risk of MI in newborns with CF

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Huntington's Disease News and Research

Latest Huntington's Disease News and Research

eBook: Advancing neurodegenerative research with iPSC-derived microglia models eBook

Shaping the Future of Neuroscience: A Conversation with Atlas Antibodies on the MolBoolean™ and the Impact of SfN 2024

How the Arts Reshape Brain Function: Susan Magsamen on the Future of Neuroaesthetics

Inside the Alzheimer's Association: Dr. Heather Snyder on Driving Research and Collaboration

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