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Transplanted hematopoietic stem cells reverse damage caused by neuro-muscular disorder
Researchers at University of California San Diego School of Medicine report that a single infusion of wildtype hematopoietic stem and progenitor cells (HSPCs) into a mouse model of Friedreich's ataxia (FA) measurably halted cellular damage caused by the degenerative disease. The findings, published online in the October 25 issue of Science Translational Medicine, suggest a potential therapeutic approach for a disease that currently is considered incurable. (Source: World Pharma News)
Source: World Pharma News - October 26, 2017 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news

Transplanted hematopoietic stem cells reverse damage caused by neuro-muscular disorder
(University of California - San Diego) Researchers at University of California San Diego School of Medicine report that a single infusion of wildtype hematopoietic stem and progenitor cells (HSPCs) into a mouse model of Friedreich's ataxia (FA) measurably halted cellular damage caused by the degenerative disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 25, 2017 Category: International Medicine & Public Health Source Type: news

Jupiter Orphan Therapeutics Receives Orphan Drug Designation for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich's Ataxia
JUPITER, Fla., Aug. 22, 2017 -- (Healthcare Sales & Marketing Network) -- Jupiter Orphan Therapeutics, Inc. ("JOT"), Jupiter, FL, today announced that it has received notification from the US Food and Drug Administration (FDA) that its Orphan Dr... Biopharmaceuticals, Neurology, FDA Jupiter Orphan Therapeutics, JOTROL, Resveratrol, Friedreich's Ataxia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - August 22, 2017 Category: Pharmaceuticals Source Type: news

Hope for Patients with Friedreich's Ataxia and Related Diseases - Neurodegenerative Disease Mechanism and Potential Drug Identified
UCD researchers report on new studies of progressive, neurodegenerative diseases linked to defects in cells mitochondria and hope for developing a new biomarker and drug for treating such diseases (Source: Disabled World)
Source: Disabled World - June 8, 2017 Category: Disability Tags: Medical Research Source Type: news

What Are the Clinical Symptoms Associated with Friedreich Ataxia?
Discussion Friedreich ataxia (FRDA) was first extensively described in a series of papers from 1863-1877 by Nikolaus Friedreich at the University of Heidelberg, Germany. In 1996 the genetic mutation was described. It is an autosomal recessively inherited, homologous expansion of the GAA repeat in intron 1 of the frataxin gene on chromosome 9q13. It causes a transcription error leading to a decrease in frataxin which is a mitochondrial protein involved in iron metabolism and other cell functions. Frataxin is seen mainly in the central and peripheral nervous systems, heart, pancreas and skeleton. Frataxin is produced but in ...
Source: PediatricEducation.org - April 10, 2017 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Geelong mother opens up about rare inherited disease
Leah Alstin, from Geelong, was diagnosed with Friedreich's ataxia when she was 17 years old. The rare inherited disease progressively damages the nervous system. (Source: the Mail online | Health)
Source: the Mail online | Health - March 2, 2017 Category: Consumer Health News Source Type: news

Horizon Pharma plc to Acquire Worldwide Rights to Interferon Gamma-1b From Boehringer Ingelheim International GmbH
Also Licenses Rights to Patents and Pending Applications Covering Methods for Treating Friedreich's Ataxia With Interferon Gamma-1b; Company Currently Owns the Rights to Interferon Gamma-1b Under the Trade Name ACTIMMUNE(R) in the United States, Canada and... Biopharmaceuticals, Licensing, AcquisitionsHorizon Pharma, Boehringer Ingelheim, interferon gamma-1b (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - May 19, 2016 Category: Pharmaceuticals Source Type: news

Horizon Pharma completes patent enrolment for Phase lll trial of actimmuneto treat Friedreich's Ataxia
Ireland-based biopharmaceutical company Horizon Pharma has completed its target enrolment of 90 patients for Phase lll trial of actimmune (interferon gamma-1b) to treat Friedreich's Ataxia (FA) disease. (Source: Drug Development Technology)
Source: Drug Development Technology - May 5, 2016 Category: Pharmaceuticals Source Type: news

UT Southwestern scientists find potential treatment for Friedreich's ataxia
(UT Southwestern Medical Center) Researchers at UT Southwestern Medical Center have identified synthetic RNA and DNA that reverses the protein deficiency causing Friedreich's ataxia, a neurological disease for which there is currently no cure. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - February 16, 2016 Category: Biology Source Type: news

University of South Florida and Friedreich's Ataxia Research Alliance to host scientific symposium
(University of South Florida (USF Health)) The University of South Florida will again bring together leading researchers and patients searching for a treatment for Friedreich's ataxia and related disorders at the seventh annual scientific symposium 'Understanding Energy for A Cure.' The symposium will be held 5:00 to 8:30 p.m., Thursday, Sept. 17, at the USF Marshall Student Center Ballroom in Tampa, FL. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 1, 2015 Category: Global & Universal Source Type: news

Horizon Pharma begins Actimmune’s Phase III trial for Friedreich's Ataxia
Ireland-based Horizon Pharma has started the Phase III study of Actimmune (interferon gamma-1b) to treat patients with Friedreich's Ataxia (FA), a degenerative neuro-muscular disorder. (Source: Drug Development Technology)
Source: Drug Development Technology - June 7, 2015 Category: Pharmaceuticals Source Type: news

Horizon Pharma's Friedreich's Ataxia Drug Gets Fast-Track Designation
Horizon Pharma PLC said Friday that its experimental Friedreich’s ataxia treatment has received fast-track designation from the U.S. Food and Drug Administration, a move that could speed approval. (Source: WSJ.com: Health)
Source: WSJ.com: Health - April 10, 2015 Category: Pharmaceuticals Tags: PAID Source Type: news

Horizon Pharma plc Receives FDA Fast Track Designation for ACTIMMUNE(R) in the Treatment of Friedreich's Ataxia
DUBLIN, IRELAND--(Healthcare Sales & Marketing Network) - Horizon Pharma plc (NASDAQ: HZNP), a specialty biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated products that a... Biopharmaceuticals, Neurology, FDAHorizon Pharma, ACTIMMUNE, Friedreich's Ataxia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 10, 2015 Category: Pharmaceuticals Source Type: news

Horizon Pharma plc Submits Investigational New Drug Application for ACTIMMUNE(R) in the Treatment of Friedreich's Ataxia
Phase 3 Study Expected to Begin in Q2 2015 DUBLIN, IRELAND--(Healthcare Sales & Marketing Network) - Horizon Pharma plc (HZNP), a specialty biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commerci... Biopharmaceuticals, Neurology, FDAHorizon Pharma, ACTIMMUNE, Friedreich's Ataxia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - February 13, 2015 Category: Pharmaceuticals Source Type: news

Gait and balance in adults with Friedreich's ataxia - Stephenson J, Zesiewicz T, Gooch C, Wecker L, Sullivan K, Jahan I, Kim SH.
Friedreich's ataxia (FA) is an autosomal recessive, neurodegenerative disease characterized by progressive muscle weakness and sensory loss, balance deficits, and gait ataxia. Gait and balance impairments become worse as the disease progresses, but limited... (Source: SafetyLit: All (Unduplicated))
Source: SafetyLit: All (Unduplicated) - February 13, 2015 Category: Global & Universal Tags: Ergonomics, Human Factors, Anthropometrics, Physiology Source Type: news

Converting Skin Cells into Sensory Neurons that Detect Body Sensations
A team led by scientists from The Scripps Research Institute (TSRI) has found a simple method to convert human skin cells into the specialized neurons that detect pain, itch, touch and other bodily sensations. These neurons are also affected by spinal cord injury and involved in Friedreich's ataxia, a devastating and currently incurable neurodegenerative disease that largely strikes children. (Source: Disabled World)
Source: Disabled World - November 24, 2014 Category: Disability Tags: Medical Research Source Type: news

Pain and itch in a dish
(Scripps Research Institute) A team led by scientists from The Scripps Research Institute has found a simple method to convert human skin cells into the specialized neurons that detect pain, itch, touch and other bodily sensations. These neurons are also affected by spinal cord injury and involved in Friedreich's ataxia, a devastating and currently incurable neurodegenerative disease that largely strikes children. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 24, 2014 Category: Global & Universal Source Type: news

Daily vitamin B3 intake could help treat degenerative childhood disease
(NaturalNews) Vitamin B3 may be a miracle treatment for the genetic childhood disease Friedreich's ataxia -- which currently has no treatment or cure -- according to a study conducted by researchers from Imperial College London, University College London and the National Institute... (Source: NaturalNews.com)
Source: NaturalNews.com - May 22, 2014 Category: Consumer Health News Source Type: news

Nicotinamide shows mixed results in patients with Friedreich ataxia
Read the full story on MD Consult: Nicotinamide shows mixed results in patients with Friedreich ataxia (Source: MD Consult: News: Top Stories)
Source: MD Consult: News: Top Stories - May 3, 2014 Category: Journals (General) Source Type: news

Friedreich's ataxia -- an effective gene therapy in an animal model
(INSERM (Institut national de la santé et de la recherche médicale)) The team led by Hélène Puccio, director of research for Inserm at the Institute of Genetics and Molecular and Cellular Biology in close collaboration with Patrick Aubourg's team has demonstrated, in the mice, the efficacy of gene therapy for treating the heart disease associated with Friedreich's ataxia, a rare hereditary neuro-degenerative disorder. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - April 6, 2014 Category: Biology Source Type: news

FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia
EPI-743 in two phase 2 development studies for Friedreich's ataxia MOUNTAIN VIEW, Calif., March 17, 2014 -- (Healthcare Sales & Marketing Network) -- Edison Pharmaceuticals today announced that the United States Food and Drug Administration granted Fas... BiopharmaceuticalsEdison Pharmaceuticals, EPI-743, Friedreich's ataxia, Vincerinone (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - March 17, 2014 Category: Pharmaceuticals Source Type: news

International patient alliance to fund Spanish Friedreich's ataxia gene-therapy project
(Institute for Research in Biomedicine (IRB Barcelona)) The gene therapy-based research project to tackle Friedreich's ataxia launched in November in labs at the Institute for Research in Biomedicine, in Barcelona, and the "Centro de Biología Molecular Severo Ochoa", in Madrid, has received 100,000 US$ per year for two years from the Friedreich's Ataxia Research Alliance. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - January 28, 2014 Category: Biology Source Type: news

Agilis and Intrexon partner to develop DNA-based therapeutics for rare genetic disease
US-based synthetic biology companies Agilis Biotherapeutics and Intrexon have entered into an exclusive channel collaboration (ECC) agreement to develop DNA-based therapeutics for Friedreich's ataxia (FRDA), a rare genetic neurodegenerative disease. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - January 3, 2014 Category: Pharmaceuticals Source Type: news

Patients and scientists join forces to tackle Friedreich's Ataxia
(Institute for Research in Biomedicine (IRB Barcelona)) The Spanish Federation of Ataxia -- in representation of the GENEFA Platform for a Friedreich's Ataxia cure --, the Babel Family association for biomedical research into Friedreich's Ataxia, the "Centro de Biología Molecular Severo Ochoa", and the Institute for Research in Biomedicine have signed an agreement through which these patients' associations will fund, by means of donations, a 3-year research project addressing Friedreich's Ataxia. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 14, 2013 Category: Global & Universal Source Type: news

What Is The Cost Of Rare Diseases Such As Friedreich's Ataxia?
What is the cost of rare diseases such as Friedreich's Ataxia? By analyzing direct and indirect costs of care research in BioMed Central's open access journal Orphanet Journal of Rare Diseases calculated that conservatively this rare disease costs between £11,000 and £19,000 per person per year. Proper understanding resource allocation is important in minimizing the effect of Friedreich's Ataxia on people's lives while maximizing their quality of life. Diseases are classified as "rare" if they affect less than 1 in 2000 people... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - March 1, 2013 Category: Consumer Health News Tags: Genetics Source Type: news

Edison commences EPI-743 Phase IIb Friedreich's ataxia trial
Edison Pharmaceuticals has commenced a Phase IIb clinical trial of EPI-743, an orally bioavailable small molecule being developed for the treatment of Friedreich's ataxia and other inherited mitochondrial diseases. (Source: Drug Development Technology)
Source: Drug Development Technology - January 8, 2013 Category: Pharmaceuticals Source Type: news