10th of June 2011
MOUNTAIN VIEW, Calif., June 10, 2011 /PRNewswire/ -- Edison Pharmaceuticals, Inc. announced today preliminary results obtained on a 28-day phase 2A clinical trial in Friedreich's ataxia.
EPI-A0001 did significantly improve neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS).
Read more
Saturday, June 11, 2011
Friday, June 10, 2011
Emergency Use Protocol for EPI-743 in Acutely Ill Patients With Inherited Mitochondrial Respiratory Chain Disease Within 90 Days of End-of-Life Care
This study is currently recruiting participants.
First Received on June 7, 2011. Last Updated on June 9, 2011
Sponsor: Edison Pharmaceuticals Inc
Information provided by: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT01370447
Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
First Received on June 7, 2011. Last Updated on June 9, 2011
Sponsor: Edison Pharmaceuticals Inc
Information provided by: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT01370447
Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Transcranial Sonography Reveals Cerebellar, Nigral, and Forebrain Abnormalities in Friedreich’s Ataxia
Neurodegenerative Dis, (DOI: 10.1159/000327751)
Matthis Synofzik, Jana Godau, Tobias Lindig, Ludger Schöls, Daniela Berg
Department of Neurodegenerative Diseases, Hertie Institute for Clinical Brain Research, and German Research Center for Neurodegenerative Diseases, University of Tübingen, Tübingen, Germany
Keywords: Ataxia, Transcranial sonography, Friedreich’s ataxia, Imaging, Substantia nigra, Dentate nucleus, Iron, Cerebellum
Matthis Synofzik, Jana Godau, Tobias Lindig, Ludger Schöls, Daniela Berg
Department of Neurodegenerative Diseases, Hertie Institute for Clinical Brain Research, and German Research Center for Neurodegenerative Diseases, University of Tübingen, Tübingen, Germany
Keywords: Ataxia, Transcranial sonography, Friedreich’s ataxia, Imaging, Substantia nigra, Dentate nucleus, Iron, Cerebellum
Mortality in Friedreich Ataxia
Journal of the Neurological Sciences,Article in Press,
doi:10.1016/j.jns.2011.05.023
(J Neurol Sci. 2011 Aug 15;307(1-2):46-9. Epub 2011 Jun 8.)
Amy Y. Tsoua, Erin K. Paulsena, b, c, d, Sarah J. Lagedrosta, b, c, d, Susan L. Perlmane, Katherine D. Mathewsf, George R. Wilmotg, Bernard Ravinah, Arnulf H. Koeppeni, j and David R. Lyncha, b, c, d,
a Department of Neurology, University of Pennsylvania Medical School, United States
b Department of Pediatrics, University of Pennsylvania Medical School, United States
c Children's Hospital of Philadelphia, United States
d University of Pennsylvania Medical School, United States
e University of California Los Angeles, Los Angeles, CA, United States
f University of Iowa, Iowa City, IA, United States
g Emory University, Atlanta, GA, United States
h University of Rochester, Rochester, NY, United States
i VA Hospital, Albany, NY, United States
Keywords: Cerebellum; Sensory; Cardiomyopathy; Echocardiogram; Arrhythmia; Neurodegenerative disease
doi:10.1016/j.jns.2011.05.023
(J Neurol Sci. 2011 Aug 15;307(1-2):46-9. Epub 2011 Jun 8.)
Amy Y. Tsoua, Erin K. Paulsena, b, c, d, Sarah J. Lagedrosta, b, c, d, Susan L. Perlmane, Katherine D. Mathewsf, George R. Wilmotg, Bernard Ravinah, Arnulf H. Koeppeni, j and David R. Lyncha, b, c, d,
a Department of Neurology, University of Pennsylvania Medical School, United States
b Department of Pediatrics, University of Pennsylvania Medical School, United States
c Children's Hospital of Philadelphia, United States
d University of Pennsylvania Medical School, United States
e University of California Los Angeles, Los Angeles, CA, United States
f University of Iowa, Iowa City, IA, United States
g Emory University, Atlanta, GA, United States
h University of Rochester, Rochester, NY, United States
i VA Hospital, Albany, NY, United States
Keywords: Cerebellum; Sensory; Cardiomyopathy; Echocardiogram; Arrhythmia; Neurodegenerative disease
Thursday, June 9, 2011
Repligen Reports Fourth Quarter ........ "RG2833 for Friedreich’s Ataxia"
9th of June 2011
RG2833 for Friedreich’s Ataxia
We are currently developing histone deacetylase class 1 inhibitors for the treatment of inherited neurodegenerative diseases such as Friedreich's ataxia. Friedreich's ataxia is caused by inadequate production of the protein frataxin which leads to degeneration of the nerves controlling muscle movements. Pending regulatory approval, we plan to initiate a Phase 1 study of RG2833 in Friedreich's ataxia patients in Europe by the end of the year. We are currently engaged in discussions with potential
pharmaceutical partners to evaluate whether partnering this program would increase the trajectory for global development and ultimately maximize the commercial potential of the asset.
RG2833 for Friedreich’s Ataxia
We are currently developing histone deacetylase class 1 inhibitors for the treatment of inherited neurodegenerative diseases such as Friedreich's ataxia. Friedreich's ataxia is caused by inadequate production of the protein frataxin which leads to degeneration of the nerves controlling muscle movements. Pending regulatory approval, we plan to initiate a Phase 1 study of RG2833 in Friedreich's ataxia patients in Europe by the end of the year. We are currently engaged in discussions with potential
pharmaceutical partners to evaluate whether partnering this program would increase the trajectory for global development and ultimately maximize the commercial potential of the asset.
Stem Cell Transplantation Offers 'Sea Of Opportunity' For Treating Debilitating Diseases
Medical News Today, Article Date: 09 Jun 2011
An article in the current issue of Technology & Innovation, Proceedings of the National Academy of Inventors ™ reports on the bright future and enormous need for stem cell therapeutics that may offer hope for those suffering from debilitating and deadly diseases. read more...
An article in the current issue of Technology & Innovation, Proceedings of the National Academy of Inventors ™ reports on the bright future and enormous need for stem cell therapeutics that may offer hope for those suffering from debilitating and deadly diseases. read more...
Edison Pharmaceuticals Announces FDA Grants EPI-743 Orphan Drug Designation
Medical News Today, Article Date: 09 Jun 2011
Edison Pharmaceuticals, Inc. announced the United States Food and Drug Administration has granted orphan drug designation to EPI-743 for treatment of inherited mitochondrial respiratory chain diseases. read more
Edison Pharmaceuticals, Inc. announced the United States Food and Drug Administration has granted orphan drug designation to EPI-743 for treatment of inherited mitochondrial respiratory chain diseases. read more
Edison Pharmaceuticals ofrecerá un acceso ampliado al EPI-743 para la enfermedad mitocondrial
MOUNTAIN VIEW, California, 8 de junio de 2011 /PRNewswire/
La FDA autorizó un Programa de Acceso Ampliado con el propósito de administrar el fármaco en fase de investigación EPI-743 a los pacientes graves diagnosticados con enfermedades mitocondriales heredadas de la cadena respiratoria. leer más...
La FDA autorizó un Programa de Acceso Ampliado con el propósito de administrar el fármaco en fase de investigación EPI-743 a los pacientes graves diagnosticados con enfermedades mitocondriales heredadas de la cadena respiratoria. leer más...
Wednesday, June 8, 2011
Edison Pharmaceuticals to Provide Expanded Access to EPI-743 for Mitochondrial Disease
7th of June 2011, Source: PR Newswire
MOUNTAIN VIEW, Calif., June 8, 2011 /PRNewswire/ -- Edison Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has allowed an Expanded Access program to provide EPI-743 to seriously ill patients diagnosed with inherited respiratory chain diseases of the mitochondria. read more
Edison Pharmaceuticals élargit l'accès à EPI-743 pour les maladies mitochondriales
MOUNTAIN VIEW, Californie, June 8, 2011 /PRNewswire/ --
- La FDA autorise l'utilisation d'un nouveau médicament expérimental pour les cas menaçant le pronostic vital
Edison Pharmaceuticals, Inc. a aujourd'hui annoncé que la "Food and Drug Administration" américaine (la FDA) a autorisé un programme d'accès élargi pour offrir EPI-743 aux patients gravement malades ayant reçu un diagnostic de maladies héréditaires de la chaîne respiratoire mitochondriale. Les patients dont la maladie a été génétiquement confirmée comme ceux qui répondent à certains critères cliniques, en l'absence de confirmation génétique, sont tous éligibles. Lire plus.......
Edison Pharmaceuticals sorgt für erweiterten Zugang zu EPI-743 für mitochondriale Erkrankungen
MOUNTAIN VIEW, Kalifornien, June 8, 2011 /PRNewswire/ --
- FDA genehmigt Anwendung von Medikament in klinischer Testphase (Investigational New Drug) bei lebensbedrohlichen Befunden
Edison Pharmaceuticals, Inc. gab heute bekannt, dass die amerikanische Zulassungsbehörde für Lebens- und Arzneimittel, die US Food and Drug Administration (FDA), ein sogenanntes Expanded-Access-Programm genehmigt hat, durch das EPI-743 an schwerkranke Patienten mit diagnostizierter Störung der Atmungskette aufgrund mitochondrialer Erbkrankheiten verabreicht werden darf. Darunter fallen sowohl Patienten mit genetisch bestätigter Krankheit als auch Patienten, die bei fehlender genetischer Bestätigung spezifische klinische Kriterien erfüllen. Lesen Sie mehr ...
MOUNTAIN VIEW, Calif., June 8, 2011 /PRNewswire/ -- Edison Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has allowed an Expanded Access program to provide EPI-743 to seriously ill patients diagnosed with inherited respiratory chain diseases of the mitochondria. read more
Edison Pharmaceuticals élargit l'accès à EPI-743 pour les maladies mitochondriales
MOUNTAIN VIEW, Californie, June 8, 2011 /PRNewswire/ --
- La FDA autorise l'utilisation d'un nouveau médicament expérimental pour les cas menaçant le pronostic vital
Edison Pharmaceuticals, Inc. a aujourd'hui annoncé que la "Food and Drug Administration" américaine (la FDA) a autorisé un programme d'accès élargi pour offrir EPI-743 aux patients gravement malades ayant reçu un diagnostic de maladies héréditaires de la chaîne respiratoire mitochondriale. Les patients dont la maladie a été génétiquement confirmée comme ceux qui répondent à certains critères cliniques, en l'absence de confirmation génétique, sont tous éligibles. Lire plus.......
Edison Pharmaceuticals sorgt für erweiterten Zugang zu EPI-743 für mitochondriale Erkrankungen
MOUNTAIN VIEW, Kalifornien, June 8, 2011 /PRNewswire/ --
- FDA genehmigt Anwendung von Medikament in klinischer Testphase (Investigational New Drug) bei lebensbedrohlichen Befunden
Edison Pharmaceuticals, Inc. gab heute bekannt, dass die amerikanische Zulassungsbehörde für Lebens- und Arzneimittel, die US Food and Drug Administration (FDA), ein sogenanntes Expanded-Access-Programm genehmigt hat, durch das EPI-743 an schwerkranke Patienten mit diagnostizierter Störung der Atmungskette aufgrund mitochondrialer Erbkrankheiten verabreicht werden darf. Darunter fallen sowohl Patienten mit genetisch bestätigter Krankheit als auch Patienten, die bei fehlender genetischer Bestätigung spezifische klinische Kriterien erfüllen. Lesen Sie mehr ...
Differential Expression of PGC-1α and Metabolic Sensors Suggest Age-Dependent Induction of Mitochondrial Biogenesis in Friedreich Ataxia Fibroblasts
2011 PLoS ONE 6(6): e20666. doi:10.1371/journal.pone.0020666, OPEN ACCESS
García-Giménez JL, Gimeno A, Gonzalez-Cabo P, Dasí F, Bolinches-Amorós A, et al.
The induction of mitochondrial biogenesis in FRDA may be a consequence of the mitochondrial impairment associated with disease evolution. The increase of ROS and the involvement of the oxidative phosphorylation may be an early event in the cell pathophysiology of frataxin deficiency, whereas increase of mitochondriogenic response might be a later phenomenon associated to the individual age and natural history of the disease, being more evident as the patient age increases and disease evolves. This is a possible explanation of heart disease in FRDA.
FULL TEXT PDF
García-Giménez JL, Gimeno A, Gonzalez-Cabo P, Dasí F, Bolinches-Amorós A, et al.
The induction of mitochondrial biogenesis in FRDA may be a consequence of the mitochondrial impairment associated with disease evolution. The increase of ROS and the involvement of the oxidative phosphorylation may be an early event in the cell pathophysiology of frataxin deficiency, whereas increase of mitochondriogenic response might be a later phenomenon associated to the individual age and natural history of the disease, being more evident as the patient age increases and disease evolves. This is a possible explanation of heart disease in FRDA.
FULL TEXT PDF
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