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 total revenue for first-quarter fiscal 2011 increases 39% to $7.01 million

Repligen Corporation today reported results for the first quarter of fiscal year 2011, ended June 30, 2010. Total revenue for the first quarter was $7,010,000 compared to total revenue of $5,061,000 for the first quarter of fiscal year 2010, an increase of $1,949,000 or 39%. Bioprocessing product revenue for the first quarter was $4,269,000 compared to $2,473,000 for the first quarter of fiscal 2010, an increase of $1,796,000 or 73%.

5 Aug 2010

Scientists identify IRP proteins to ensure iron balance for cell survival

Most organisms need iron to survive, but too much iron is toxic, and can cause fatal organ failure. The same is true inside cells, where iron balance must also be maintained. In a study published today in Cell Metabolism, scientists at the European Molecular Biology Laboratory (EMBL) in Heidelberg, Germany, have discovered that a group of proteins named IRPs ensure that this iron balance is kept and as such are essential for cell survival. More specifically, they found that IRPs are required for the functioning of mitochondria, the cell's energy factories.

4 Aug 2010

Repligen receives three additional grants to develop HDAC inhibitors for neurodegenerative diseases

Repligen Corporation today announced that the Company has received three additional grants to support its research program to develop HDAC inhibitors for neurodegenerative diseases. Repligen has received $436,000 in research funding to support the ongoing development of histone deacetylase inhibitors for Friedreich's ataxia.

2 Aug 2010

Repligen's fiscal 2010 total revenue decreases to $20,971,000

Repligen Corporation today reported results for the fourth quarter and fiscal year 2010, ended March 31, 2010. Total revenue for the year was $20,971,000 compared to total revenue of $29,362,000 for fiscal year 2009 ended March 31, 2009.

9 Jun 2010

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Repligen Corporation announced today that the Office of Orphan Products Development of the Food and Drug Administration has granted orphan drug designation to RG2833, a selective histone deacetylase 3 inhibitor for the treatment of Friedreich's ataxia. Orphan drug designation qualifies Repligen to receive seven years of marketing exclusivity in the United States if the company is the first to obtain marketing approval for RG2833 for the treatment of Friedreich's ataxia.

24 May 2010

Tufts University's School of Arts and Sciences receives $9.5M grant to create research space

Tufts University's School of Arts and Sciences has received a $9.5 million grant to create research space that will house a Collaborative Cluster in Genome Structure and Developmental Patterning in Health and Disease.

17 May 2010

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