Friedreich’s Ataxia News and Research

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Preliminary results from Edison's EPI-A0001 28-day phase 2A trial against Friedreich's ataxia

Edison Pharmaceuticals, Inc. announced today preliminary results obtained on a 28-day phase 2A clinical trial in Friedreich's ataxia. While the primary endpoint of glucose disposition index did not show statistically significant improvement, EPI-A0001 did significantly improve neurological function as assessed by the Friedreich's Ataxia Rating Scale.

10 Jun 2011

Repligen total revenue increases 30% to $27,291,000 for fiscal year 2011

Repligen Corporation today reported results for the fourth quarter and fiscal year 2011, ended March 31, 2011.

9 Jun 2011

Scripps Research receives CIRM grant for new research in stem cell biology and disease origins

Stem cells have huge potential in medicine because they have the ability to change or differentiate into many different cell types potentially providing a source of cells to replace those that have been permanently lost by a patient.

6 May 2011

Repligen anticipates 30% increase in total revenues for 2011

Repligen Corporation today announced preliminary financial results for fiscal year 2011 which ended March 31, 2011. Total revenue for the year is expected to be $27.3 million compared to $21.0 million in the prior fiscal year, an increase of 30%.

21 Apr 2011

Top-line results from Repligen RG2417 Phase 2b trial results in patients with bipolar depression

Repligen Corporation announced today results of a Phase 2b clinical trial of RG2417, an oral formulation of uridine, in patients with bipolar depression.

8 Mar 2011

Repligen third quarter total revenue increases 26% to $7,068,000

Repligen Corporation today reported results for the third quarter of fiscal year 2011, ended December 31, 2010. Total revenue for the quarter was $7,068,000 compared to total revenue of $5,617,000 for the third quarter of fiscal year 2010 ended December 31, 2009, an increase of $1,451,000 or 26%.

9 Feb 2011

MDA awards Repligen $1.4M for research of new compound with therapeutic potential for SMA

The Muscular Dystrophy Association today announced it has awarded $1.4 million in new research funding to the biopharmaceutical firm Repligen of Waltham, Mass., to complete the preclinical work needed to begin human clinical trials of a promising therapeutic compound for spinal muscular atrophy (SMA).

17 Dec 2010

MDA supports Repligen with $1.4M grant for RG3039 Spinal Muscular Atrophy development program

Repligen Corporation today announced that the Company has received $1,400,000 in research funding from the Muscular Dystrophy Association ("MDA") to support the ongoing development of RG3039 for Spinal Muscular Atrophy ("SMA"). RG3039, our lead compound, is an inhibitor of an RNA processing enzyme which targets increased production of SMN, a protein of deficient levels in patients with SMA.

15 Dec 2010

New findings could help scientists devise optimal therapies for Friedreich's ataxia

Scripps Research Institute scientists have taken a step closer to understanding the cause of Friedreich's ataxia, a debilitating neurological condition that affects tens of thousands of people worldwide, and so far has no cure.

8 Nov 2010

Muscular Dystrophy Association awards $14.1M in new grants

The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants.

20 Aug 2010

MDA awards $14.1 million in new grants to advance treatments and cures for muscle diseases

18 Aug 2010

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

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