Huntington's Disease News and Research

Latest Huntington's Disease News and Research

Anti-seizure drug can be used to treat AT deficiency: Pitt researchers

The liver scarring of α1-antitrypsin deficiency, the most common genetic cause for which children undergo liver transplantation, might be reversed or prevented with a medication that has long been used to treat seizures, according to findings from Children's Hospital of Pittsburgh of UPMC and the University of Pittsburgh School of Medicine that will published in Science and are available online today through the Science Express website.

4 Jun 2010

Raptor Pharmaceutical acquires exclusive license to intellectual property for Huntington's Disease treatment

Raptor Pharmaceutical Corp., announced that the Company has acquired an exclusive worldwide license to intellectual property related to the potential treatment of Huntington's Disease from the Weizmann Institute of Science in Israel and Niigata University in Japan. In addition, Raptor has added Professor Lawrence Steinman, M.D., an inventor on the Weizmann patent, to its Advisory Board.

2 Jun 2010

Alnylam, Mount Sinai scientists discover svRNAs that play a critical role in influenza A virus replication

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the publication of new research findings in the journal Proceedings of the National Academy of Sciences (PNAS) by Alnylam scientists and collaborators from Mount Sinai School of Medicine. In the new study, Alnylam and Mount Sinai scientists discovered a novel class of virus-produced small non-coding RNAs, called small viral RNAs (svRNAs), which play a critical role in the replication of influenza A virus.

2 Jun 2010

Neuralstem updates progress of ongoing Phase I human clinical trial for ALS

Neuralstem, Inc. updated the progress of its ongoing Phase I human clinical trial to treat ALS (Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease) at Emory University in Atlanta, Georgia. The company announced that, after reviewing the safety data from the first cohort of three patients, the Safety Monitoring Board has approved moving to the next cohort and transplantation of the fourth patient.

25 May 2010

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Repligen Corporation announced today that the Office of Orphan Products Development of the Food and Drug Administration has granted orphan drug designation to RG2833, a selective histone deacetylase 3 inhibitor for the treatment of Friedreich's ataxia. Orphan drug designation qualifies Repligen to receive seven years of marketing exclusivity in the United States if the company is the first to obtain marketing approval for RG2833 for the treatment of Friedreich's ataxia.

24 May 2010

Scientists identify new gene candidates that regulate aggregate formations in Huntington's disease

They might not be known for their big brains, but fruit flies are helping to make scientists and doctors smarter about what causes Huntington's disease and how to treat it. New research, published in the journal GENETICS (http://www.genetics.org) describes a laboratory test that allows scientists to evaluate large numbers of fruit fly genes for a possible role in the formation of plaque-like protein aggregates within cells.

24 May 2010

EPO rules in favor of Alnylam Pharmaceuticals’ Kreutzer-Limmer '945 patent

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the Board of Appeals of the European Patent Office (EPO) has ruled that claims of the Kreutzer-Limmer '945 (EP 1214945) patent meet needed requirements under European patent law. The '945 patent comprises broad claims covering the length and certain structural requirements for siRNAs that are important for advancement of RNAi therapeutics.

20 May 2010

Study confirms inverse association of increase in BMI with lower brain volumes

Researchers from Boston University School of Medicine determined that excess abdominal fat places otherwise healthy, middle-aged people at risk for dementia later in life. Preliminary findings suggest a relationship between obesity and dementia that could lead to promising prevention strategies in the future. Results of this study are published early online in Annals of Neurology, a journal of the American Neurological Association.

20 May 2010

Neurologix reports lower net loss of $3.5M for first-quarter 2010

Neurologix, Inc., a biotechnology company developing innovative gene therapies for the brain and central nervous system, today announced its financial results for the quarter ended March 31, 2010.

14 May 2010

Being physical and mental activity delays onset of Huntington’s disease: Research

Melbourne researchers report in a study that regular physical and mental activities can delay the onset of the dreaded genetic Huntington's disease. This disease is hereditary and it goes on to destroy the brain and nerve cells leading to loss of all or most faculties. It affects one in 10,000 people between ages 30 and 50 and there is no cure. Since it is passed on to the children at least 50% of the time the decision to have children also becomes an agonizing one for many sufferers.

13 May 2010

Repligen files IND for Phase 1 study of RG2833 selective HDAC-3 inhibitor for Friedreich's ataxia

Repligen Corporation announced today that it has filed an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) for a Phase 1 study of RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor. This is a double-blind, single ascending dose, Phase 1 study in healthy volunteers to evaluate the pharmacokinetic and safety profile of RG2833 in up to 40 subjects.

13 May 2010

Key mechanisms for delivery of LNP-encapsulated siRNAs to the liver

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the publication of pre-clinical research in the journal Molecular Therapy revealing key mechanisms related to the systemic delivery of RNAi therapeutics using lipid nanoparticles (LNPs).

12 May 2010

Evotec first-quarter operating loss decreases 93% to € 1.5 m

Evotec AG today reported financial results and corporate updates for the first quarter of 2010.

12 May 2010

Neuroscientist Snyder presents annual Ellis S. Grollman Lecture at UMB

If a drug was developed to block a key protein linked to the onset of Huntington's Disease, it could have a clear path to the part of the brain most affected by the disease, while not bothering other parts of the brain and body, said distinguished neuroscientist Solomon H. Snyder, MD, to an audience of about 300 students and faculty at the University of Maryland, Baltimore (UMB).

6 May 2010

Alnylam Pharmaceuticals reports consolidated financial results for first quarter 2010

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2010, and company highlights.

6 May 2010

AMRI first-quarter total revenue down 9% to $49.3 million

AMRI today reported financial and operating results for the first quarter ended March 31, 2010.

5 May 2010

Alnylam Biotherapeutics presents new data on RNAi technologies at Cell Culture Engineering XII

Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the presentation of new data at the Cell Culture Engineering XII conference held in Alberta, Canada from April 25 - 30, 2010. Alnylam Biotherapeutics was formed by Alnylam to develop RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies.

4 May 2010

Alnylam Pharmaceuticals announces EPO intent to grant notification for patent series covering RNAi therapeutics

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the European Patent Office (EPO) has issued a notification of an intent to grant for the "Manoharan II" patent series (EP Application No 04 718 537), titled "Therapeutic Compositions." The new patent includes 16 claims covering compositions and methods, including pharmaceutical compositions, for chemically modified siRNAs containing phosphorothioate and 2′-O-alkyl modifications without any siRNA length restrictions.

3 May 2010

Raptor Pharmaceutical presents positive results from Phase 2a trial of DR cysteamine bitartrate for NASH

Raptor Pharmaceutical Corp., announced positive Phase 2a clinical trial results from its pilot study of delayed-release cysteamine bitartrate in 11 adolescent patients with non-alcoholic steatohepatitis, a progressive form of liver disease believed to affect 5% to 11% of the U.S. population. The results were presented at the Digestive Disease Week 2010 conference in New Orleans, LA on May 2, 2010.

3 May 2010

Neuralstem's stem cell treatment for ALS featured on CNN with Dr. Sanjay Gupta

Neuralstem, Inc. announced that its stem cell treatment for ALS (Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease), currently in a FDA-approved Phase I clinical trial, was featured on CNN last night with Dr. Sanjay Gupta, in the piece entitled "Stem Cell Medical Breakthrough".

30 Apr 2010

Huntington's Disease News and Research

Latest Huntington's Disease News and Research

Anti-seizure drug can be used to treat AT deficiency: Pitt researchers

Raptor Pharmaceutical acquires exclusive license to intellectual property for Huntington's Disease treatment

Alnylam, Mount Sinai scientists discover svRNAs that play a critical role in influenza A virus replication

Neuralstem updates progress of ongoing Phase I human clinical trial for ALS

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Scientists identify new gene candidates that regulate aggregate formations in Huntington's disease

EPO rules in favor of Alnylam Pharmaceuticals’ Kreutzer-Limmer '945 patent

Study confirms inverse association of increase in BMI with lower brain volumes

Neurologix reports lower net loss of $3.5M for first-quarter 2010

Being physical and mental activity delays onset of Huntington’s disease: Research

Repligen files IND for Phase 1 study of RG2833 selective HDAC-3 inhibitor for Friedreich's ataxia

Key mechanisms for delivery of LNP-encapsulated siRNAs to the liver

Evotec first-quarter operating loss decreases 93% to € 1.5 m

Neuroscientist Snyder presents annual Ellis S. Grollman Lecture at UMB

Alnylam Pharmaceuticals reports consolidated financial results for first quarter 2010

AMRI first-quarter total revenue down 9% to $49.3 million

Alnylam Biotherapeutics presents new data on RNAi technologies at Cell Culture Engineering XII

Alnylam Pharmaceuticals announces EPO intent to grant notification for patent series covering RNAi therapeutics

Raptor Pharmaceutical presents positive results from Phase 2a trial of DR cysteamine bitartrate for NASH

Neuralstem's stem cell treatment for ALS featured on CNN with Dr. Sanjay Gupta

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

Flow chemistry, AI, and NMR: A next-gen approach to scalable API production

Digital pathology and the PathoVerse: A conversation with Dr. Singh

Bioprocess Efficiency with Cell Density Monitoring

Huntington's Disease News and Research

Latest Huntington's Disease News and Research

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

Flow chemistry, AI, and NMR: A next-gen approach to scalable API production

Digital pathology and the PathoVerse: A conversation with Dr. Singh

Bioprocess Efficiency with Cell Density Monitoring

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