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Archive for August 2010

GSF’s Fundraising Campaign for Gene Therapy Research Reaches Milestone

Posted: August 30th, 2010 | By: Staff | No Comments
Monday, August 30th, 2010

This summer, FightSMA and the Gwendolyn Strong Foundation (GSF) partnered in an effort to raise funding for spinal muscular atrophy (SMA) gene therapy research.

GSF’s goal is to raise $200,000 before the end of 2010. Today, they announced that they have reached the halfway mark.

From GSF:

GSF $200 for SMA badgeLess than TWO months after launching our “$200K For SMA” fundraising campaign in support of promising SMA focused gene therapy research at Nationwide Children’s Hospital and The Ohio State University, friends, families, colleagues, coworkers, communities, and total strangers across the country – some impacted by SMA and many not – have come together to organize and execute an impressive laundry list of wonderful fundraisers totaling over – DRUM ROLL PLEASE – One Hundred Thousand Dollars. That’s right. $100,000!!! In…just…TWO…months.

There’s just no other way to say it – what’s happening here is incredible, inspiring, and humbling. And we are personally honored to be part of all of this positivity – it fuels us to push forward and do more. At our core, we’ve always believed in the power of the individual. And as individuals, if we collectively work together in a positive, productive fashion, we can have an enormous impact and change the status quo.

Click here to read the full announcement.

A huge “THANK YOU!” goes out to everyone who has been involved in raising funds for GSF’s “$200K for SMA” (read the list here) and FightSMA’s “Realizing the Dream” – campaigns worked together towards the same goal: to bring SMA gene therapy to clinical trial.

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Categories : Spinal Muscular Atrophy Families and Friends, Spinal Muscular Atrophy Science and Research

Article addresses the difficulties of developing new therapies

Posted: August 26th, 2010 | By: Staff | No Comments
Thursday, August 26th, 2010

The web magazine, Slate, has a thought-provoking article entitled “The Medical Revolution: Where are the cures promised by stem cells, gene therapy, and the human genome?” which addresses the difficulties of developing new therapies.

To read the article, click here.

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Categories : Spinal Muscular Atrophy Science and Research

Possible funding opportunities for SMA researchers

Posted: August 24th, 2010 | By: Staff | No Comments
Tuesday, August 24th, 2010

The following are three National Institutes of Health (NIH) funding opportunities that may be of interest to researchers working toward a treatment and cure for spinal muscular atrophy (SMA), a deadly crippler and the number-one inherited genetic cause of infant death.

Exceptional, Unconventional Research Enabling Knowledge Acceleration (EUREKA) (R01)
Application Due Date: October 21, 2010
Solicits proposals for exceptionally innovative research on novel hypotheses or difficult problems, solutions to which would have an extremely high impact on biomedical or biobehavioral research. Support may be requested for up to $800,000 in direct costs (excluding consortium F&A) over a four-year period, not exceeding $250,000 (direct costs, excluding consortium F&A) in any one year. (Note, that over the last three years the EUREKA mechanism has had a success rate much more in line with standard R01 applications. Each of these offers an opportunity not as easily addressed in regular NIH funding mechanisms.)

NIH Common Fund Transformative Research Projects Program (R01)
Application Due Date(s): October 27, 2010
Solicits applications proposing groundbreaking, exceptionally innovative, high risk, original and/or unconventional research with the potential to create new scientific paradigms or challenge existing ones. Budget requests should be commensurate with project needs for up to a five-year project period. Up to one third of the budget for this FOA will be reserved projects exceeding $1 million dollars in direct costs.

Scalable Assays for Unbiased Analysis of Neurobiological Function (R01)
Application Due Date: November 17, 2010
Solicits applications to develop novel, robust analytical platforms using in vitro assays to reveal changes in neuronal and/or glial function. The goal is to adapt state-of-the-art measures of basic cellular processes or molecular events that are key mediators of brain function with the intent to probe mechanisms and/or perturbations in an unbiased and efficient manner. The novel assay platforms would provide opportunities to measure neurobiological endpoints and build a pipeline to be used in the context of target identification and drug discovery.

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Categories : Spinal Muscular Atrophy Science and Research

SMA to be represented at the 2010 SfN meeting

Posted: August 23rd, 2010 | By: Staff | No Comments
Monday, August 23rd, 2010

The Society for Neuroscience (SfN) is the world’s largest organization of scientists and physicians devoted to advancing understanding of the brain and nervous system. SfN’s 40th annual meeting, Neuroscience 2010, will be held November 13-17 in San Diego, California. As in past years, the exhibit hall will have an “SMA Organizations” booth devoted to raising awareness of spinal muscular atrophy (SMA) among the researchers and scientists attending the meeting.

In addition, Families of SMA, FightSMA, MDA, and SMA Foundation will be sponsoring a satellite event. Entitled “Nucleic Acids to the Rescue: Gene and Antisense Oligonucleotide Therapies for SMA,” the symposia meeting will be held Monday, November 15th at the Manchester Grand Hyatt, just a block from the SfN meeting site. (This event is not sponsored by the Society for Neuroscience.)

Click on the image below to view the save the date.

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Categories : Spinal Muscular Atrophy Science and Research

More studies show concern about cardiac defects in SMA patients

Posted: August 19th, 2010 | By: Staff | No Comments
Thursday, August 19th, 2010

Work in the Lorson lab at the University of Missouri, the DiDonato lab at Northwestern University, and the Kaspar lab at Nationwide Children’s Hospital have recently shown functional and developmental cardiac defects in two severe models of spinal muscular atrophy (SMA). The studies were conducted with mouse models, not SMA patients, and all three were recently published in the journal Human Molecular Genetics.

According to Dr. Chris Lorson, FightSMA’s Science Director and also an author of one of the studies, “collectively these results highlight the importance of additional tissues in SMA that may contribute to the overall pathology in SMA – most likely in more severe cases. This is especially important when developing and testing potential therapeutics in SMA models and as novel compounds progress towards the clinic.”

Click the links below to read the article abstracts:

  • Lorson Lab: Cardiac defects contribute to the pathology of spinal muscular atrophy models
  • DiDonato Lab: Arrhythmia and cardiac defects are a feature of spinal muscular atrophy model mice
  • Kaspar Lab: Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery
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Categories : General Information
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