Pengfei Lin, Junling Liu, Ming Ren, Kunqian Ji, Ling Li, Bin Zhang, Yaoqin Gong, Chuanzhu Yan, Idebenone Protects against Oxidized Low Density Lipoprotein Induced Mitochondrial Dysfunction in Vascular Endothelial Cells via GSK3β/β-catenin signaling pathways, Biochemical and Biophysical Research Communications, Available online 15 August 2015, ISSN 0006-291X, http://dx.doi.org/10.1016/j.bbrc.2015.08.058.
Monday, August 17, 2015
Intel just open sourced Stephen Hawking’s speech system and it’s a .NET 4.5 WinForms app that you can try for yourself
Mansib Rahman, Canadian Developer Connection. 14 Aug 2015
As we all know, the venerable physicist Professor Stephen Hawking is unable to talk as he is afflicted with ALS and thus relies on a computer system to communicate. In 2011, his condition was deteriorating so badly that he could best communicate at a rate of only 2 words per minute. He reached out to Alan Moore at Intel and asked if Intel could come up with new technology to help his plight.
Well, what are you waiting for? You can try all this out for yourself. Visit the software's Github release page to get the installer.
As we all know, the venerable physicist Professor Stephen Hawking is unable to talk as he is afflicted with ALS and thus relies on a computer system to communicate. In 2011, his condition was deteriorating so badly that he could best communicate at a rate of only 2 words per minute. He reached out to Alan Moore at Intel and asked if Intel could come up with new technology to help his plight.
Well, what are you waiting for? You can try all this out for yourself. Visit the software's Github release page to get the installer.
Nrf2—a therapeutic target for the treatment of neurodegenerative diseases
Nrf2—a therapeutic target for the treatment of neurodegenerative diseases. Delinda A. Johnson, Jeffrey A. Johnson, Free Radical Biology and Medicine, Available online 14 August 2015, Page FRBMD1500535, ISSN 0891-5849, http://dx.doi.org/10.1016/j.freeradbiomed.2015.07.147.
The brain is very sensitive to changes in redox status; thus maintaining redox homeostasis in the brain is critical for the prevention of accumulating oxidative damage. Recently, a clinical trial using RTA 408 was initiated for Friedreich’s ataxia, a neurodegenerative condition responsible for cerebellar ataxia due to impaired production of the protein frataxin leading to profound deficiencies in mitochondrial respiration (ClinicalTrials.gov Identifier NCT02255435).
The brain is very sensitive to changes in redox status; thus maintaining redox homeostasis in the brain is critical for the prevention of accumulating oxidative damage. Recently, a clinical trial using RTA 408 was initiated for Friedreich’s ataxia, a neurodegenerative condition responsible for cerebellar ataxia due to impaired production of the protein frataxin leading to profound deficiencies in mitochondrial respiration (ClinicalTrials.gov Identifier NCT02255435).
Wednesday, August 12, 2015
PATENT: SMALL MOLECULE ACTIVATORS OF MITOCHONDRIAL FUNCTION
NEW PATENT: SMALL MOLECULE ACTIVATORS OF MITOCHONDRIAL FUNCTION.
Inventor(s): WILSON ROBERT B [US]; COTTICELLI MARIA GRAZIA [US]; BENEDETTI PHILLIP A [US]; SMITH AMOS [US]; MELVIN JASON E [US]; HURYN DONNA M [US]
Applicant(s): UNIV PENNSYLVANIA [US]
Original document: WO2010068767 (A1) ― 2010-06-17
Inventor(s): WILSON ROBERT B [US]; COTTICELLI MARIA GRAZIA [US]; BENEDETTI PHILLIP A [US]; SMITH AMOS [US]; MELVIN JASON E [US]; HURYN DONNA M [US]
Applicant(s): UNIV PENNSYLVANIA [US]
Original document: WO2010068767 (A1) ― 2010-06-17
Saturday, August 8, 2015
Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia
Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia. BUSINESS WIRE, August 05, 2015
The patent broadly protects a promising method for treating cardiomyopathy by using an adeno-associated virus (AAV) vector to carry into cells a gene expressing the protein frataxin. The patent will run until 2033 or longer in the event of a successful application for an extension. Corresponding patent applications are pending in major markets globally.
The patent broadly protects a promising method for treating cardiomyopathy by using an adeno-associated virus (AAV) vector to carry into cells a gene expressing the protein frataxin. The patent will run until 2033 or longer in the event of a successful application for an extension. Corresponding patent applications are pending in major markets globally.
Monday, August 3, 2015
The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review
The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review. Y. Schuller, C. E. M. Hollak and M. Biegstraaten; Orphanet Journal of Rare Diseases 2015, 10:92 doi:10.1186/s13023-015-0305-y
OPEN ACCESS
In the European Union (EU), a disease is considered ‘orphan’ if it is a life-threatening or seriously debilitating disorder that affects fewer than 1 per 2 000. An orphan disease is defined in the EU as a disorder affecting less than 1 in 2 000 individuals. The concept of ultra-orphan has been proposed for diseases with a prevalence of less than 1:50 000. According to this classification Friedreich's Ataxia is within the group of the "orphan", although is close to the upper border of the "ultra-orphan", so share with the "ultra-orphan" many of the problems for the development of drugs and therapies.
OPEN ACCESS
In the European Union (EU), a disease is considered ‘orphan’ if it is a life-threatening or seriously debilitating disorder that affects fewer than 1 per 2 000. An orphan disease is defined in the EU as a disorder affecting less than 1 in 2 000 individuals. The concept of ultra-orphan has been proposed for diseases with a prevalence of less than 1:50 000. According to this classification Friedreich's Ataxia is within the group of the "orphan", although is close to the upper border of the "ultra-orphan", so share with the "ultra-orphan" many of the problems for the development of drugs and therapies.
Friday, July 31, 2015
Friedreich Ataxia in Classical Galactosaemia
Friedreich Ataxia in Classical Galactosaemia. Siobhán Neville, Siobhan O’Sullivan, Bronagh Sweeney, Bryan Lynch, Donncha Hanrahan, Ina Knerr, Sally Ann Lynch, Ellen Crushell; JIMD Reports, 29 Jul 2015, DOI 10.1007/8904_2015_477
Both conditions are known to occur with increased frequency amongst the Irish Traveller population. Neurological symptoms are easily attributed to an underlying diagnosis of galactosaemia. It is important to consider a diagnosis of Friedreich ataxia in a child from the Irish Traveller population with galactosaemia who presents with ataxia or cardiomyopathy.
Both conditions are known to occur with increased frequency amongst the Irish Traveller population. Neurological symptoms are easily attributed to an underlying diagnosis of galactosaemia. It is important to consider a diagnosis of Friedreich ataxia in a child from the Irish Traveller population with galactosaemia who presents with ataxia or cardiomyopathy.
Friedreich’s Ataxia Research Collaboration Announced
Friedreich’s Ataxia Research Collaboration Announced. Medical Sciences Division, University of Oxford, 30 July 2015.
A new collaborative drug discovery project in Friedreich’s Ataxia (FA) between the University of Oxford, Ataxia UK, Pfizer Inc, UCL and Imperial College London was recently announced.
The programme will initially run for three years and aims to develop a potential new medicine or therapy for Friedreich’s ataxia that, if successful, may be tested in clinical trials.
A new collaborative drug discovery project in Friedreich’s Ataxia (FA) between the University of Oxford, Ataxia UK, Pfizer Inc, UCL and Imperial College London was recently announced.
The programme will initially run for three years and aims to develop a potential new medicine or therapy for Friedreich’s ataxia that, if successful, may be tested in clinical trials.
Tuesday, July 28, 2015
Les médicaments orphelins : des opportunités méconnues pour les développeurs en Europe
Les médicaments orphelins : des opportunités méconnues pour les développeurs en Europe. Orphan Drugs: Underrated Opportunities for The Developers in Europe. Yves Tillet et Anne-Catherine Maillols-Perroy; Thérapie 2015 Juillet-Août; 70 (4): 351–357
Key words: Orphan Drug Act / regulation 141/2000/EC / implementing regulation 847/2000 / Commission communication (2003/C 178/02) / orphan medical product / 10 year market exclusivity / similar medicinal product / significant benefit / clinical superiority / assumption of significant benefit
Key words: Orphan Drug Act / regulation 141/2000/EC / implementing regulation 847/2000 / Commission communication (2003/C 178/02) / orphan medical product / 10 year market exclusivity / similar medicinal product / significant benefit / clinical superiority / assumption of significant benefit
Sunday, July 26, 2015
Mitigation of Myocardial Ischemia-Reperfusion Injury via HIF-1α-Frataxin Signalling.
Mitigation of Myocardial Ischemia-Reperfusion Injury via HIF-1α-Frataxin Signalling. Nelson Amaral, Darlington Okonko; American Journal of Physiology - Heart and Circulatory Physiology Published 25 July 2015 Vol. no. , DOI: 10.1152/ajpheart.00553.2015
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