Sunday, October 26, 2014

Fitting and verification of frequency modulation systems on children with normal hearing.

Fitting and verification of frequency modulation systems on children with normal hearing. Schafer, Erin C.; Bryant, Danielle; Sanders, Katie; Baldus, Nicole; Algier, Katherine; Lewis, Audrey; Traber, Jordan; Layden, Paige; Amin, Aneeqa; Source: Journal of the American Academy of Audiology, Volume 25, Number 6, June 2014, pp. 529-540(12)

Several recent investigations support the use of frequency modulation (FM) systems in children with normal hearing and auditory processing or listening disorders such as those diagnosed with auditory processing disorders, autism spectrum disorders, attention-deficit hyperactivity disorder, Friedreich ataxia, and dyslexia.

The Role of the Cerebellum in Cognition Beyond Coordination in the Central Nervous System

The Role of the Cerebellum in Cognition Beyond Coordination in the Central Nervous System; Maryam Noroozian, Volume 32, Issue 4, November 2014, Pages 1081–1104 DOI: 10.1016/j.ncl.2014.07.005

This article reviews in further detail the current understanding of cognitive deficits associated with cerebellar impairments and unravels its modulating role in cognitive and behavioral processes.

Neuro-ophthalmic Manifestations of Cerebellar Disease

Neuro-ophthalmic Manifestations of Cerebellar Disease; Shin C. Beh , Teresa C. Frohman , Elliot M. Frohman; Neurologic Clinics, Volume 32, Issue 4 DOI: 10.1016/j.ncl.2014.07.002

The cerebellum is responsible for refining ocular movements, thereby guaranteeing the best possible visual acuity and clarity despite changes in body or head positions or movement of the object of interest.

The Cerebellum and Sleep

The Cerebellum and Sleep; Lourdes M. DelRosso, Romy Hoque; Neurologic Clinics; Volume 32, Issue 4, November 2014, Pages 893–900 DOI: 10.1016/j.ncl.2014.07.003

Sleep medicine is an important and under-recognized part of the neurologic evaluation in those with cerebellar disease.

Thursday, October 23, 2014

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models. Catherine Gérard, Xiao Xiao, Mohammed Filali, Zoé Coulombe, Marie Arsenault, Jacques Couet, Juan Li, Marie-Claude Drolet, Pierre Chapdelaine, Amina Chikh & Jacques P Tremblay; Molecular Therapy — Methods & Clinical Development 1, Article number: 14044 ​doi:10.1038/mtm.2014.44

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Wednesday, October 22, 2014

Open-label pilot study of interferon gamma-1b in Friedreich ataxia

Open-label pilot study of interferon gamma-1b in Friedreich ataxia. L. Seyer, N. Greeley, D. Foerster, C. Strawser, S. Gelbard, Y. Dong, K. Schadt, M. G. Cotticelli, A. Brocht, J. Farmer, R. B. Wilson and D. R. Lynch; Acta Neurol Scand: DOI: 10.1111/ane.12337.

Conclusions – Interferon gamma-1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo-controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN-c in FRDA.

Thursday, October 16, 2014

FA & ACTIMMUNE Phase 2 Clinical Trial Results

FA & ACTIMMUNE Phase 2 Clinical Trial Results. Friedreich's Ataxia Analyst Day Presentation, Horizon Pharma plc, October 13, 2014

Clinical Potentials of Cardiomyocytes Derived from Patient-Specific Induced Pluripotent Stem Cells

Clinical Potentials of Cardiomyocytes Derived from Patient-Specific Induced Pluripotent Stem Cells. Kwong-Man Ng, Cheuk-Yiu Law and Hung-Fat Tse; J. Clin. Med. 2014, 3(4), 1105-1123; doi:10.3390/jcm3041105

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The cardiomyocytes derived from patients-specific iPSCs are of great potential in many clinical applications. This authentic human cardiomyocyte-based system is expected to compensate for the limitations of the current experimental animal models. This review provides detailed descriptions in the strategies and workflow of using the patient-specific iPSCs-derived cardiomyocytes in regenerative medicine, disease modeling and pharmacological applications. The examples illustrated in this review clearly evidenced the practical values of this novel technology. However, various limitations, such as the immaturities of iPSCs-derived cardiomyocytes, still need to be addressed, and future studies resolving these issues would be beneficial to the use of patient-specific iPSCs in clinical applications.

Monday, October 13, 2014

Horizon Pharma plc Reviews Future ACTIMMUNE® Opportunities at Analyst Day

Horizon Pharma plc Reviews Future ACTIMMUNE® Opportunities at Analyst Day. KING OF PRUSSIA, PA--(Marketwired - Oct 13, 2014)

SOURCE: Horizon Pharma plc