Spinal Muscular Atrophy, the #1 genetic killer of children under the age of two

[FayeforCure]

Mom in the Spotlight
Victoria Strong

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Pictured: Victoria, Bill, and Gwendolyn Strong



Victoria & Bill are the parents of a beautiful 9 month old girl, Gwendolyn.  Gwendolyn has a disease called Type 1 Spinal Muscular Atrophy, the #1 genetic killer of children under the age of two.  It has no treatment, no cure.  Proud parents Victoria and Bill keep a website updated with the latest information (and cute photos) chronicling their daughter's life.



When they learned of a bill before Congress (H.R. 3334/S. 2042) to help fund organizations that are striving to find treatments and cures for SMA, the Strongs took action and created an online petition to encourage Congress to pass this legislation.  Their goal was 1,000 signatures, but they reached that goal so quickly that thier revised goal is now 10,000 signatures. 

Victoria kindly allowed SBParent.com to feature her so that we may learn a little more about her.  While this might not be appropriately called a "Fun Q&A" (Sorry for the default wording I cannot change), this is certainly a phenomenal woman that is doing everything she can to make a difference.

http://www.sbparent.com/Moms_on_the_Move/index.html?id=20


Excellent way to save these babies:

CSC's stem cell-derived motor neuron product for SMAreceives FDA orphan drug designation
December 2009 05:27

California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that the FDA has granted orphan drug designation to MOTORGRAFTâ,,¢, a stem cell-derived motor neuron product, for the treatment of Spinal Muscular Atrophy (SMA).

Orphan drug designation, granted by the FDA Office of Orphan Products Development, provides several incentives to companies in the private sector developing novel drugs or biologics to treat diseases with relatively small market potential. These include seven years market exclusivity following FDA approval, clinical trial design assistance, reduced user fees and tax credits.

CSC developed MOTORGRAFTâ,,¢ as a stem cell-derived motor neuron replacement product for the treatment of SMA. Pre-clinical GLP safety and efficacy studies, funded significantly by the Families of Spinal Muscular Atrophy and conducted by Professor Hans S. Keirstead of the University of California at Irvine, have demonstrated safety and functional benefit in several animal models.

SMA, the leading genetic killer of infants, causes the deterioration of the muscles that control crawling, walking, swallowing and breathing and affects approximately 1 in 6000 babies born. In the US approximately 7.5 million people (1 in 40) are carriers of the defective gene. There are no currently approved therapies for the treatment of SMA.

CSC recently completed a formal pre-IND meeting with the FDA to discuss the clinical and regulatory pathway for submission of an application to initiate human trials using this therapy for the treatment of SMA Type I. The company expects to file an IND to begin a Phase I safety study in 2010.

“We are extremely encouraged to receive orphan-drug designation for MOTORGRAFTâ,,¢,” said Chris Airriess, CSC’s Chief Operating Officer, “this is a major milestone in realizing our commitment to find a treatment for SMA.”

“Families of SMA is pleased to see this promising therapy advancing towards clinical trials,” said Jill Jarecki, Research Director for FSMA, “Orphan Designation will provide CSC with all the opportunities afforded by Orphan Drug Act of 1983, which is intended to facilitate a close working relationship between regulatory agencies and companies with the aim of accelerating the drug development and approval processes for rare diseases.”

SOURCE California Stem Cell, Inc.

http://www.news-medical.net/news/20091209/CSCs-stem-cell-derived-motor-neuron-product-for-SMA-receives-FDA-orphan-drug-designation.aspx

http://uniteforthecure.com/sma-stem-cell-program/

SMA is twice as common as ALS and is the leading genetic killer of children under the age of 2.

Stem Cell Research Center co-director Hans Keirstead spearheaded the project, in which embryonic stem cells were implanted in animals and coaxed into becoming working motor nerves.

"To date, there has been no method of isolating high-purity cultures, 95 percent and up, where we can generate unlimited quantities of this tissue type," he said.

Those nerve tissues could be used as replacement therapies for ALS, SMA, MS and spinal-cord injuries.

http://www.dailybulletin.com/ci_5998837?source=most_emailed

Save the SMA Babies!!!