Saturday, November 7, 2015

Patent: METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO FRIEDREICH ATAXIA

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Inventor(s): PUCCIO HELENE MONIQUE [FR]; AUBOURG PATRICK [FR]; CRYSTAL RONALD G [US]; BOUGNERES PIERRE [FR] Application number: US20151471...

Management of Children with Mild, Moderate, and Moderately Severe Sensorineural Hearing Loss

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Anne Marie Tharpe, Samantha Gustafson, Otolaryngologic Clinics of North America, Volume 48, Issue 6, December 2015, Pages 983-994, ISSN 0030...
Friday, November 6, 2015

UAB researchers seek Friedreich’s ataxia biomarkers

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UAB News, University of Alabama at Birmingham, November 04, 2015 “There is a high demand for biomarkers because of ongoing clinical trial...

Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich's Ataxia Study

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ClinicalTrials.gov Identifier: NCT02593773, First received: October 29, 2015  The purpose of this phase 3 multi-center, open-label extens...

Frataxin Is Localized to Both the Chloroplast and Mitochondrion and Is Involved in Chloroplast Fe-S Protein Function in Arabidopsis.

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Turowski VR, Aknin C, Maliandi MV, Buchensky C, Leaden L, Peralta DA, Maria V. Busi, Alejandro Araya, Diego F. Gomez-Casati, PLoS ONE 10(10...

A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?

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Oo, C. and Rusch, L. M. Journal of Clinical Pharma. doi: 10.1002/jcph.599 Nevertheless, there is no better opportunity than the present to...
Tuesday, October 27, 2015

Fronto-cerebellar dysfunction and dysconnectivity underlying cognition in friedreich ataxia: The IMAGE-FRDA study

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Ian H. Harding1, Louise A. Corben, Elsdon Storey, Gary F. Egan, Monique R. Stagnitti, Govinda R. Poudel, Martin B. Delatycki and Nellie Geor...
Tuesday, October 20, 2015

URINARY, BOWEL AND SEXUAL FUNCTION IN PATIENTS WITH FRIEDREICH'S ATAXIA

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Meher Lad, Michael Parkinson, Myriam Rai, Massimo Pandolfo, Sinead Murphy, Anton Emmanuel, Jalesh Panicker, Paola Giunti; J Neurol Neurosurg...
Monday, October 19, 2015

Compassionate use of orphan drugs

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Hanna I. Hyry, Jeremy Manuel, Timothy M. Cox and Jonathan C. P. Roos; Orphanet Journal of Rare Diseases 2015, 10:100 doi:10.1186/s13023-015...
Sunday, October 18, 2015

Patient-Funded Trials: Opportunity or Liability?

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Danielle Marie Wenner, Jonathan Kimmelman, Alex John London, Patient-Funded Trials: Opportunity or Liability?, Cell Stem Cell, Volume 17, Is...