Ataxia News and Research

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Ataxia is a neurological sign and symptom that consists of gross lack of coordination of muscle movements. Ataxia is a non-specific clinical manifestation implying disfunction of the parts of the nervous system that coordinate movement, such as the cerebellum.

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

Researchers examine expression of toxic mRNA in patients with Fragile X Tremor Ataxia Syndrome

Expression of a toxic RNA that leads to Fragile X Tremor Ataxia Syndrome is modifiable by genetic or pharmacologic means, according to new research from U-M Medical School scientists.

15 Dec 2010

Study finds expression of toxic RNA that leads to Fragile X Tremor Ataxia Syndrome modifiable

Expression of a toxic RNA that leads to Fragile X Tremor Ataxia Syndrome is modifiable by genetic or pharmacologic means, according to new research from U-M Medical School scientists.

14 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

Researchers find gene mutation mechanism that cause mitochondrial diseases

Scientists have discovered a new cause of spastic ataxia, and believe this cause is also a trigger for other mitochondrial diseases neurological disorders that can lead to serious coordination, growth, visual, speech, and muscle defects.

22 Oct 2010

FDA grants clearance for HPβCD treatment for Niemann Pick Type C disease

Children's Hospital & Research Center Oakland announced today that the US Food and Drug Administration (FDA) has granted clearance of an Investigational New Drug (IND) application to introduce Trappsol Cyclo (Hydroxypropyl Beta Cyclodextrin or HPβCD) into the brains of six year old identical twin girls dying of a rare brain-destroying cholesterol disease called Niemann Pick Type C (NPC).

24 Sep 2010

H1N1 influenza increases neurological complications in children: Study

A recent study by researchers at the University of Utah determined that the 2009 pandemic influenza A (H1N1) caused a higher rate of neurological complications in children than the seasonal flu. The most common complications observed were seizures and encephalopathy.

20 Sep 2010

Ataxin 2 gene mutation contributes to Lou Gehrig's disease: Research

An international study led by biologists and neuroscientists from the University of Pennsylvania has identified a new genetic risk factor for amyotrophic lateral sclerosis, commonly known as ALS or Lou Gehrig's disease.

26 Aug 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

Asuragen reports collaborative study results of advanced PCR-based molecular profiling of FMR1 gene

Asuragen, Inc. announced today the results from two collaborative studies, one with the University of California Davis M.I.N.D. Institute and another with Rush University Medical Center, that demonstrate comprehensive molecular profiling of the Fragile X Mental Retardation (FMR1) gene using advanced PCR-based methods.

26 Jul 2010

Research on new treatments for FXS to be presented at International Fragile X Conference

Accelerated research findings leading to potentially significant new treatments for fragile X syndrome will be presented to nearly 1,000 family members and professionals at the 12th International Fragile X Conference in Dearborn July 21-25.

23 Jul 2010

Researchers identify a key player in dramatic loss of neurons in mice and fly models

A team of researchers, led by scientists at the University of California, San Diego, have identified a key player in the dramatic loss of neurons in mice and fly models, a discovery that could help illuminate the role of mitochondrial dysfunction in human neurodegenerative disorders, such as Parkinson's disease.

25 Jun 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

Discovery reveals a new approach to target cancer

Researchers at the University of Pittsburgh Cancer Institute (UPCI) and the School of Medicine have discovered that inhibiting a key molecule in a DNA repair pathway could provide the means to make cancer cells more sensitive to radiation therapy while protecting healthy cells.

3 Jun 2010

Motion Therapeutics to launch BalanceWear Stabilizing Garments for MS

For those who suffer from balance disorders and loss of mobility, promising results from a new stabilizing device may help to reestablish mobility, confidence and independence. This week, Motion Therapeutics premieres its BalanceWear™ Stabilizing Garments at the 24th Annual Meeting for the Consortium of Multiple Sclerosis Centers in San Antonio.

2 Jun 2010