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.

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

USPTO grants new patent to Intellect's Alzheimer's vaccine

Intellect Neurosciences, Inc., a biopharmaceutical company with an internal preclinical and clinical-stage pipeline and licenses with major pharmaceutical companies covering products in late-stage clinical trials, announced today that it has received a new patent from the United States Patent and Trademark Office in relation to the Company's RECALL-VAXTM technology platform.

9 Mar 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

Scientists develop Australia's first pluripotent stem cell lines using skin biopsies for FA disease

Scientists have developed Australia's first adult induced pluripotent stem cell lines using skin biopsies from patients with the rare genetic disease Friedreich Ataxia.

14 Jan 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

Santhera, Ipsen partner to develop and commercialize fipamezole outside North America and Japan

Santhera Pharmaceuticals and Ipsen today announced a license agreement for the development and commercialization of fipamezole (antagonist of the adrenergic alpha-2 receptor) for territories outside of North America and Japan. This first-in-class compound is currently under investigation for the treatment of levodopa-induced dyskinesia in Parkinson's Disease.

3 Sep 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

Penwest Pharmaceuticals signs merger agreement with Endo Pharmaceuticals

Penwest Pharmaceuticals Co. today announced that it has entered into a merger agreement under which its long-time partner in the development and commercialization of Opana ER, Endo Pharmaceuticals, has agreed to acquire all of the common stock of Penwest for $5.00 per share in cash. The total equity value of the transaction is approximately $168 million.

10 Aug 2010

Endo to acquire Penwest for $5.00 per share

Endo Pharmaceuticals today announced actions designed to advance the company's leadership and growth in pain management, including an agreement to acquire all outstanding shares of Penwest Pharmaceuticals for $5.00 in cash per share, or an estimated enterprise value of approximately $144 million at the time of deal close. Penwest has been working with Endo since 1997 on the development and commercialization of OPANA® ER and receives a royalty stream on net sales of the product.

10 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