Horizon Pharma plc Submits Investigational New Drug Application for ACTIMMUNE® in the Treatment of Friedreich's Ataxia
. Marketwired, February 13, 2015
SOURCE: Horizon Pharma plc
Phase 3 Study Expected to Begin in Q2 2015
Friday, February 13, 2015
PIPs in neurological diseases
PIPs in neurological diseases. M.G. Waugh, Biochim. Biophys. Acta (2015), http://dx.doi.org/10.1016/j.bbalip.2015.02.002
There has been an interesting development recently concerning the possible involvement of PIP5K1B in Friedreich's ataxia.
There has been an interesting development recently concerning the possible involvement of PIP5K1B in Friedreich's ataxia.
Thursday, February 12, 2015
Thérapie génique : 3 millions d’euros pour AAVLife
Thérapie génique : 3 millions d’euros pour AAVLife. Bpifrance 12 février 2015
Gene therapy for FA is blooming, yesterday knew the good news of the major strategic collaboration between Sanofi-Genzyme and Voyager Therapeutics, today it has known the news of a new economic aportacion to AAVLIFE project, which develops a gene therapy with AAV vectors for FA, targeted to the heart
Bpifrance investit, via le Fonds Biothérapies innovantes et Maladies rares, 3 millions d’euros dans la société de thérapie génique AAVLife. Une opération va lui permettre de faire progresser la thérapie génique pour le traitement des cardiomyopathies liées à l’ataxie de Friedreich.
Related news:
AAVLife is a gene therapy company focused on developing treatments for rare diseases with great unmet medical need.
AAVLife Raises $12 Million in Series A Financing to Advance Gene Therapy for Friedreich’s Ataxia
Gene therapy for FA is blooming, yesterday knew the good news of the major strategic collaboration between Sanofi-Genzyme and Voyager Therapeutics, today it has known the news of a new economic aportacion to AAVLIFE project, which develops a gene therapy with AAV vectors for FA, targeted to the heart
Bpifrance investit, via le Fonds Biothérapies innovantes et Maladies rares, 3 millions d’euros dans la société de thérapie génique AAVLife. Une opération va lui permettre de faire progresser la thérapie génique pour le traitement des cardiomyopathies liées à l’ataxie de Friedreich.
Related news:
AAVLife is a gene therapy company focused on developing treatments for rare diseases with great unmet medical need.
AAVLife Raises $12 Million in Series A Financing to Advance Gene Therapy for Friedreich’s Ataxia
Voyager Therapeutics & Genzyme Announce Major Strategic Collaboration to Develop and Commercialize Novel AAV Gene Therapies for Patients with CNS Disorders
Voyager Therapeutics & Genzyme Announce Major Strategic Collaboration to Develop and Commercialize Novel AAV Gene Therapies for Patients with CNS Disorders. (BUSINESS WIRE) February 11, 2015.
Sanofi embarks on an $845M gene therapy R&D odyssey with Voyager
Up to $845 Million Collaboration to Support Development, Expansion and Commercialization of Voyager’s Novel CNS Product Pipeline
Press Release (PDF): Voyager Therapeutics & Genzyme Announce Major Strategic Collaboration to Develop and Commercialize Novel AAV Gene Therapies for Patients with CNS Disorders.
Sanofi embarks on an $845M gene therapy R&D odyssey with Voyager
Up to $845 Million Collaboration to Support Development, Expansion and Commercialization of Voyager’s Novel CNS Product Pipeline
Press Release (PDF): Voyager Therapeutics & Genzyme Announce Major Strategic Collaboration to Develop and Commercialize Novel AAV Gene Therapies for Patients with CNS Disorders.
Tuesday, February 10, 2015
Long term follow up of cardiomyopathy in Friedreich Ataxia
Long term follow up of cardiomyopathy in Friedreich Ataxia. (25es Journées Européennes de la Société Française de Cardiologie – 14-17 janvier 2015, Paris) Lise Legrand, Françoise Pousset, Sophie Tezenas du Montcel, Marie Lorraine Monin, Alina Tataru, Michel Komajda, Alexandra Durr, Richard Isnard; Archives of Cardiovascular Diseases Supplements, Volume 7, Issue 1, January 2015, Page 27, ISSN 1878-6480, http://dx.doi.org/10.1016/S1878-6480(15)71565-X.
GAA repeats are the best predictors of survival and cardiac events in FRDA patients, but EF and LV mass are also predictors of long term evolution
GAA repeats are the best predictors of survival and cardiac events in FRDA patients, but EF and LV mass are also predictors of long term evolution
Sunday, February 8, 2015
Mitochondrial iron homeostasis and its dysfunctions in neurodegenerative disorders
Mitochondrial iron homeostasis and its dysfunctions in neurodegenerative disorders. Natalia P. Mena, Pamela J. Urrutia, Fernanda Lourido, Carlos M. Carrasco, Marco T. Núñez, Mitochondrion, Available online 7 February 2015, ISSN 1567-7249, http://dx.doi.org/10.1016/j.mito.2015.02.001.
Keywords: Mitochondrial iron homeostasis; iron-sulfur cluster; heme; reactive oxygen species; neurodegenerative disease
Keywords: Mitochondrial iron homeostasis; iron-sulfur cluster; heme; reactive oxygen species; neurodegenerative disease
Milestones in Friedreich ataxia: more than a century and still learning
Milestones in Friedreich ataxia: more than a century and still learning. Agessandro Abrahão, José Luiz Pedroso, Pedro Braga-Neto, Edson Bor-Seng-Shu, Patricia de Carvalho Aguiar, Orlando Graziani Povoas Barsottini; Springer Berlin Heidelberg 2015-02-08 DOI 10.1007/s10048-015-0439-z
Keywords: Friedreich ataxia, FRDA, Autosomal recessive ataxia, Frataxin gene, FXN, Coenzyme Q10, Idebenone, Deferiprone, Erythropoietin
Keywords: Friedreich ataxia, FRDA, Autosomal recessive ataxia, Frataxin gene, FXN, Coenzyme Q10, Idebenone, Deferiprone, Erythropoietin
Friday, February 6, 2015
Una “ambulancia” para el cerebro
Una “ambulancia” para el cerebro. IRB Barcelona Scientic News
Junto a investigadores clínicos, están preparando terapias para el glioblastoma –el cáncer cerebral más agresivo en adultos-, la Ataxia de Friedreich –una enfermedad neurodegenerativa hereditaria- y próximamente para un tipo de cáncer cerebral infantil.
Junto a investigadores clínicos, están preparando terapias para el glioblastoma –el cáncer cerebral más agresivo en adultos-, la Ataxia de Friedreich –una enfermedad neurodegenerativa hereditaria- y próximamente para un tipo de cáncer cerebral infantil.
An “ambulance” for the brain
An “ambulance” for the brain. IRB Barcelona Scientific News
Together with clinical researchers, they are preparing treatments for glioblastoma—the most aggressive brain cancer in adults—, Friedreich’s Ataxia—a hereditary neurodegenerative disease—, and a type of paediatric brain cancer
Together with clinical researchers, they are preparing treatments for glioblastoma—the most aggressive brain cancer in adults—, Friedreich’s Ataxia—a hereditary neurodegenerative disease—, and a type of paediatric brain cancer
Applying the Retro-Enantio Approach to Obtain a Peptide Capable of Overcoming the Blood–Brain Barrier
Applying the Retro-Enantio Approach to Obtain a Peptide Capable of Overcoming the Blood–Brain Barrier. Dr. Roger Prades, Benjamí Oller-Salvia, Dr. Susanne M. Schwarzmaier, Dr. Javier Selva, Dr. María Moros, Matilde Balbi, Dr. Valeria Grazú, Dr. Jesus M. de La Fuente, Prof. Gustavo Egea, Prof. Nikolaus Plesnila, Dr. Meritxell Teixidó and Prof. Ernest Giralt; Angewandte Chemie Intl Edition (February 2015) DOI: 10.1002/anie.201411408
"In conclusion, we report a protease-resistant peptide with the capacity to transport cargos of distinct sizes and types across the BBB, one of the most restrictive barriers in the human body.
This potential BBB shuttle brings with it the possibility to fulfil an unmet clinical need, namely the treatment of CNS disorders."
"In conclusion, we report a protease-resistant peptide with the capacity to transport cargos of distinct sizes and types across the BBB, one of the most restrictive barriers in the human body.
This potential BBB shuttle brings with it the possibility to fulfil an unmet clinical need, namely the treatment of CNS disorders."
Subscribe to:
Posts (Atom)
