Clin. Invest. 120(1): 29-40 (2010). doi:10.1172/JCI40543. (January 4, 2010)
Olle Lindvall, Zaal Kokaia
Keywords: Stem cell–based approaches, potential treatments, neurodegenerative disorders, improve function, remyelination, trophic actions,modulation of inflammation.
Tuesday, January 5, 2010
Thursday, December 31, 2009
Subcellular targeting strategies for drug design and delivery
Nature Reviews Drug Discovery 9, 29-42 (January 2010) | doi:10.1038/nrd2897
&
drug targets, drug design strategies, bioavailability, tissue targeting, specific intracellular compartments.
Tuesday, December 29, 2009
Molecular diagnostics: techniques and applications for the clinical laboratory
Book review.
Wayne Grody, UCLA School of Medicine, Los Angeles, CA, USA Professor Divisions of Medical Genetics and Molecular Pathology Departments of Path. & Lab. Medicine, Pediatrics, and Human Genetics UCLA School of Medicine Los Angeles, CA 90095-1732
Robert Nakamura, Scripps Clinic, La Jolla, CA, U.S.A.
Frederick Kiechle, Medical Director, Clinical Pathology Department of Pathology Pathology Consultants of South Broward Memorial Healthcare System
Charles Strom, Medical Director, Genetic Testing Center Nichols Institute, Quest Diagnostics, San Juan Capistrano, CA, U.S.A. s
Hardbound, 736 pages, publication date: DEC-2009
ISBN-13: 978-0-12-369428-7
ISBN-10: 0-12-369428-0
Imprint: ACADEMIC PRESS
Wayne Grody, UCLA School of Medicine, Los Angeles, CA, USA Professor Divisions of Medical Genetics and Molecular Pathology Departments of Path. & Lab. Medicine, Pediatrics, and Human Genetics UCLA School of Medicine Los Angeles, CA 90095-1732
Robert Nakamura, Scripps Clinic, La Jolla, CA, U.S.A.
Frederick Kiechle, Medical Director, Clinical Pathology Department of Pathology Pathology Consultants of South Broward Memorial Healthcare System
Charles Strom, Medical Director, Genetic Testing Center Nichols Institute, Quest Diagnostics, San Juan Capistrano, CA, U.S.A. s
Hardbound, 736 pages, publication date: DEC-2009
ISBN-13: 978-0-12-369428-7
ISBN-10: 0-12-369428-0
Imprint: ACADEMIC PRESS
In Vivo Fluorescent Detection of Fe-S Clusters Coordinated by Human GRX2
Kevin G. Hoff1, 2, 4,, Stephanie J. Culler1, 4, Peter Q. Nguyen2, Ryan M. McGuire3, Jonathan J. Silberg2, 3, and Christina D. Smolke1
1 Division of Chemistry and Chemical Engineering, California Institute of Technology, 1200 E. California Boulevard, Mail Code 210-41, Pasadena, CA 91125, USA
2 Department of Biochemistry and Cell Biology, Rice University, 6100 Main Street, MS 140, Houston, TX 77005, USA
3 Department of Bioengineering, Rice University, 6100 Main Street, MS 140, Houston, TX 77005, USA
Keywords: Fe-S cluster biosynthesis, fluorescent approach, 2Fe2S clusters, Venus fluorescent protein, glutaredoxin 2 (GRX2), ISCU, NFS1.
1 Division of Chemistry and Chemical Engineering, California Institute of Technology, 1200 E. California Boulevard, Mail Code 210-41, Pasadena, CA 91125, USA
2 Department of Biochemistry and Cell Biology, Rice University, 6100 Main Street, MS 140, Houston, TX 77005, USA
3 Department of Bioengineering, Rice University, 6100 Main Street, MS 140, Houston, TX 77005, USA
Keywords: Fe-S cluster biosynthesis, fluorescent approach, 2Fe2S clusters, Venus fluorescent protein, glutaredoxin 2 (GRX2), ISCU, NFS1.
Splitting Fluorescent Protein Helps Image Clusters in Live Cells
AZoM.com Pty.Ltd
Posted December 24th, 2009
Keywords: Iron-sulfur clusters, Friedreich's ataxia, sideroblastic anemia, myopathy, fluorescent protein, GRX2, iron-sulfur irregularities, American Heart Association, Friedreich's Ataxia Research Alliance, Robert A. Welch Foundation.
Posted December 24th, 2009
Keywords: Iron-sulfur clusters, Friedreich's ataxia, sideroblastic anemia, myopathy, fluorescent protein, GRX2, iron-sulfur irregularities, American Heart Association, Friedreich's Ataxia Research Alliance, Robert A. Welch Foundation.
Friday, December 25, 2009
A structural and functional homolog supports a general role for frataxin in cellular iron chemistry
Communication:
Chem. Commun.2010, DOI: 10.1039/b911975bWenbin Qi and J. A. Cowan
Keyword: Bacillus subtilis, YdhG, frataxin family, iron homeostasis.
Tuesday, December 22, 2009
Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia
This study is currently recruiting participants.
Verified by Penwest Pharmaceuticals Co., December 2009
First Received: December 17, 2009 Last Updated: December 18, 2009 History of Changes
| Sponsor: | Penwest Pharmaceuticals Co. | ||
|---|---|---|---|
| Information provided by: | Penwest Pharmaceuticals Co. | ||
| ClinicalTrials.gov Identifier: | NCT01035671 |
| Condition | Intervention | Phase |
|---|---|---|
| Friedreich's Ataxia | Drug: alpha-tocopherolquinone (A0001) Drug: placebo | Phase II |
Sunday, December 20, 2009
Age and Dietary Iron Affect Expression of Genes Involved in Iron Acquisition and Homeostasis in Young Pigs.
J Nutr. 2009 Dec 16. [Epub ahead of print]
Department of Animal Science, North Carolina State University, Raleigh, NC 27695-7621.
Highlight: "frataxin decreased with age of the animal", " the expression of some genes examined in this study was affected by age".
Saturday, December 19, 2009
Coenzyme q10 in neuromuscular and neurodegenerative disorders.
Current drug targets. 2010 Jan;11(1):111-21.
Department of Neuroscience, Neurological Clinic, University of Pisa, Italy, Via Roma 67, 56126 Pisa, Italy
Keywors: Coenzyme Q10 (CoQ10, or ubiquinone), electron transport chain, oxidative phosphorylation, cellular antioxidant protection, energy metabolism, antioxidant status, neuromuscular and neurodegenerative disorders, idebenone, mitochondrial disorders, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, Friedreich's ataxia, clinical practice.
Wednesday, December 16, 2009
No difference in between-country variability in use of newly approved orphan and non- orphan medicinal products - a pilot study
Pieter Stolk , Harald E Heemstra , Hubert GM Leufkens , Brigitte Bloechl-Daum and Eibert R Heerdink
Orphanet Journal of Rare Diseases 2009, 4:27doi:10.1186/1750-1172-4-27
Published: 14 December 2009
OPEN ACCESS
Background
Regulators and payers have to strike a balance between the needs of the patient and the optimal allocation of resources. Drugs indicated for rare diseases (orphan medicines) are a special group in this context because of their often high per unit costs. Our objective in this pilot study was to determine, for drugs used in an outpatient setting, how utilisation of centrally authorised drugs varies between countries across a selection of EU member states.
Methods
We randomly selected five orphan medicines and nine other drugs that were centrally authorised in the European Union between January 2000 and November 2006. We compared utilisation of these drugs in six European Union member states: Austria, Denmark, Finland, Portugal, The Netherlands, and Sweden. Utilisation data were expressed as Defined Daily Doses per 1000 persons per year. Variability in use across countries was determined by calculating the relative standard deviation for the utilisation rates of individual drugs across countries.
Results
No association between orphan medicine status and variability in use across countries was found (P=0.52). Drugs with an orphan medicine status were more expensive and had a higher innovation score than drugs without an orphan medicine status.
Conclusions
The results show that the variability in use of orphan medicines in the different health care systems of the European Union appears to be comparable to the other newly authorised drugs that were included in the analysis. This means that, although strong heterogeneity in access may exist, this heterogeneity is not specific for drugs with an orphan status.
Full Text pdf
Orphanet Journal of Rare Diseases 2009, 4:27doi:10.1186/1750-1172-4-27
Published: 14 December 2009
OPEN ACCESS
Background
Regulators and payers have to strike a balance between the needs of the patient and the optimal allocation of resources. Drugs indicated for rare diseases (orphan medicines) are a special group in this context because of their often high per unit costs. Our objective in this pilot study was to determine, for drugs used in an outpatient setting, how utilisation of centrally authorised drugs varies between countries across a selection of EU member states.
Methods
We randomly selected five orphan medicines and nine other drugs that were centrally authorised in the European Union between January 2000 and November 2006. We compared utilisation of these drugs in six European Union member states: Austria, Denmark, Finland, Portugal, The Netherlands, and Sweden. Utilisation data were expressed as Defined Daily Doses per 1000 persons per year. Variability in use across countries was determined by calculating the relative standard deviation for the utilisation rates of individual drugs across countries.
Results
No association between orphan medicine status and variability in use across countries was found (P=0.52). Drugs with an orphan medicine status were more expensive and had a higher innovation score than drugs without an orphan medicine status.
Conclusions
The results show that the variability in use of orphan medicines in the different health care systems of the European Union appears to be comparable to the other newly authorised drugs that were included in the analysis. This means that, although strong heterogeneity in access may exist, this heterogeneity is not specific for drugs with an orphan status.
Full Text pdf
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