Friedreich's Ataxia News and Research

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Friedreich's ataxia is an inherited disease that causes progressive damage to the nervous system resulting in symptoms ranging from gait disturbance and speech problems to heart disease. A well-known celebrity afflicted with Friedreich's ataxia is former major leaguer and noted historian Clinton Stewart.

Repligen files IND for Phase 1 study of RG2833 selective HDAC-3 inhibitor for Friedreich's ataxia

Repligen Corporation announced today that it has filed an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) for a Phase 1 study of RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor. This is a double-blind, single ascending dose, Phase 1 study in healthy volunteers to evaluate the pharmacokinetic and safety profile of RG2833 in up to 40 subjects.

13 May 2010

PAREXEL further expands presence in China with offices in Chengdu and Guangzhou

PAREXEL International Corporation, a leading global biopharmaceutical services provider, today announced that the Company has opened offices in Chengdu and Guangzhou, China to support a growing requirement for a broad range of clinical development and regulatory consulting capabilities in the country and throughout the Asia/Pacific region.

From Parexel 13 May 2010

Aeterna Zentaris posts net loss of $5.9 million for first-quarter 2010

Aeterna Zentaris Inc., a late-stage drug development company specialized in oncology and endocrinology, today reported financial and operating results as at and for the three months ended March 31, 2010.

13 May 2010

Deficiency of frataxin protein causes Friedreich's ataxia

The defective gene responsible for Friedreich's ataxia was identified over 20 years ago, but so far our ideas of how this gene causes the disease have been merely speculative. It was assumed that, as a result of the mutation, the gene could no longer be transcribed because this stretch of DNA was inaccessible. FMI Group Leader Marc Bühler and his team have now carried out experiments demonstrating that this is not the case and revealing what actually happens in Friedreich's ataxia.

4 May 2010

Penwest Pharmaceuticals reports net income of $3.9 million for first-quarter 2010

Penwest Pharmaceuticals Co. today announced its financial results for the first quarter ended March 31, 2010. Compared with the first quarter of 2009, first quarter 2010 revenues increased sharply to $8.8 million, and operating expenses decreased by 20%. The Company had net income of $0.12 per share for the first quarter of 2010.

3 May 2010

Recent business progress at Penwest Pharmaceuticals

Penwest Pharmaceuticals Co. today sent a letter to its shareholders to provide an update on the Company's recent progress and on corporate governance developments.

24 Apr 2010

Repligen expects total revenue of $21.0M for fiscal year 2010

Repligen Corporation today announced preliminary financial results for fiscal year 2010 which ended March 31, 2010. Total revenue for the year is expected to be $21.0 million, including $10.3 million of bioprocessing product revenue and $10.7 million of royalty and other revenue. Research and development expenses are expected to be between $13.8-$14.2 million and selling, general and administrative expenses between $6.8-$7.2 million.

16 Apr 2010

MDA awards over $21M in grants for advancing critical neuromuscular research

Forty-two medical researchers and their labs have been awarded more than $21 million in grants by the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multi-year awards to be dispersed over the next three years.

15 Apr 2010

MDA, FFSHR award $200,000 grant for developing FSH dystrophy therapy

The Muscular Dystrophy Association (MDA), headquartered in Tucson, Ariz., and Friends of FSH Research (FFSHR), based in Kirkland, Wash., has jointly awarded a two-year, $200,000 grant to Dr. Joel Chamberlain, a research assistant professor of medical genetics at the University of Washington. The grant, equally funded by the two organizations, will enable the laboratory led by Dr. Chamberlain to study RNA interference as an investigative and therapeutic tool for facioscapulohumeral muscular dystrophy.

7 Apr 2010

MDA awards 42 medical researchers and labs over $21M for critical neuromuscular research

Forty-two medical researchers and their labs have been awarded over $21 million by the Muscular Dystrophy Association to continue critical neuromuscular research. Many of the grants are multi-year awards to be dispersed over the next three years.

1 Apr 2010

Muscular dystrophy: $1M grant for study of erectile dysfunction drugs in improving muscle blood flow

A Cedars-Sinai Heart Institute cardiologist has been awarded a three-year, nearly $1 million grant from the Muscular Dystrophy Association (MDA) to study whether drugs used to treat erectile dysfunction could also be used to improve muscle blood flow and reduce fatigue in muscular dystrophy patients.

26 Mar 2010

Cardiologist receives grant to study impact of erectile dysfunction drugs on muscular dystrophy patients

25 Mar 2010

PhRMA President honors Ron and Raychel Bartek for driving awareness of Friedreich's ataxia

Pharmaceutical Research and Manufacturers of America President and CEO Billy Tauzin today honored Ron and Raychel Bartek for their dedicated work driving awareness of Friedreich's ataxia, a debilitating, life-threatening, rare disease.

19 Mar 2010

NAF to hold third annual Ataxia Investigators Meeting in Rosemont

The National Ataxia Foundation, in conjunction with the University of Chicago program in pathobiology and translational neuroscience, will hold its third annual Ataxia Investigators Meeting March 9-11, 2010, at the Hyatt Regency O'Hare, in Rosemont, Ill. A three-day meeting for patients, caregivers and families will follow at the same location.

6 Mar 2010

PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently.

20 Jan 2010

Findings could pave way for new therapeutic approaches for Friedreich's ataxia

Elevated levels of DNA damage have for the first time been found in the cellular mitochondria and nuclei of patients with the inherited, progressive nervous system disease called Friedreich's ataxia (FRDA), says a multicenter research team led by an expert from the University of Pittsburgh Cancer Institute (UPCI).

18 Jan 2010

Test tube experiments show slight changes in protein chemistry can eliminate Huntington's disease in mice

In Huntington's disease, a mutated protein in the body becomes toxic to brain cells. Recent studies have demonstrated that a small region adjacent to the mutated segment plays a major role in the toxicity. Two new studies supported by the National Institutes of Health show that very slight changes to this region can eliminate signs of Huntington's disease in mice.

28 Dec 2009

Repligen receives $731,000 to support ongoing development of new treatments for Friedreich's ataxia

Repligen Corporation today announced that the Company has received $731,000 in research funding to support the ongoing development of new treatments for Friedreich's ataxia from the Muscular Dystrophy Association (MDA).

16 Dec 2009

New approach to protein misfolding may lead to novel therapies for a number of diseases

In an encouraging new development, a team led by Scripps Research Institute scientists has restored partial function to lung cells collected from patients with cystic fibrosis. While there is still much work to be done before the therapy can be tested in humans, the discovery opens the door to a new class of therapies for this and a host of other chronic diseases.

7 Dec 2009

Second-quarter fiscal 2010 results announced by Repligen

Repligen Corporation today reported results for the second quarter of fiscal year 2010, ended September 30, 2009. Total revenue for the quarter was $5,421,000 compared to total revenue of $5,090,000 for the second quarter of fiscal year 2009 ended September 30, 2008.

5 Nov 2009