Victoria and Bill Strong describe their daughter Gwendolyn as “a bright, happy little girl with a fighting spirit.” That spirit serves her well as she battles type 1 spinal muscular atrophy. To advance the fight against this terrible disease, the Strong family began a letter writing campaign and then created a petition to show members of Congress the support that exists for the SMA Treatment Acceleration Act.

From the Strong Family’s call to action:

PLEASE SIGN THIS PETITION TO HELP CURE SPINAL MUSCULAR ATROPHY, THE #1 GENETIC KILLER OF CHILDREN UNDER THE AGE OF 2.

We need your help to move landmark legislation through Congress that will allocate federal resources to non-profit and research organizations focused on finding a treatment and/or cure for SMA.

• SMA is an inherited genetic disease that results in loss of nerves in the spinal cord and weakness of the muscles connected with those nerves.
• SMA is the #1 genetic killer of children under the age of 2.
• SMA is estimated to occur in nearly 1 out of every 6,000 births.
• The gene mutation that causes SMA is carried by 1 in every 40 people or nearly 7.5 million American.
• There is currently no cure, but the National Institutes of Health (NIH) and the National Institute of Neurological Disorders and Stroke (NINDS) have selected SMA as the disease closest to treatment of more than 600 neurological disorders.
• Researchers estimate that we are as close as only a few years away from finding a treatment and/or cure.

Our precious daughter, Gwendolyn (http://www.GwendolynStrong.com), was born perfectly healthy in October 2007 and diagnosed with SMA at the age of 6 months. SMA is a degenerative disease that destroys the nerves controlling voluntary muscle movement, which affects crawling, walking, breathing, head and neck control, and even swallowing. Gwendolyn has Type 1 SMA, which is the most aggressive, terminal form of the disease. Gwendolyn’s mind, heart, and spirit are no different from any other baby, but her body is failing her. We will most likely lose our little girl to this disease before she reaches the age of 2.

Gwendolyn is one of thousands of children coping with this devastating disease. In fact, 600 new babies will be born in the United States with SMA this year alone. The good news is hope is on the horizon. The National Institute of Health (NIH) and the National Institute of Neurological Disorders and Stroke (NINDS) have selected SMA as the disease closest to treatment of more than 600 neurological disorders and researchers estimate that we are as close as only a few years away from finding a treatment and/or cure for SMA. However, funding is needed to make that last and crucial leap. THAT’S WHERE WE NEED YOUR HELP!!!

For the first time, legislation has been proposed in the United States Congress to allocate federal resources to non-profit and research organizations focused solely on finding a treatment and/or cure for SMA. The SMA Treatment Acceleration Act (H.R. 3334/S. 2042) was introduced in the House of Representatives as H.R. 3334 by Rep. Patrick Kennedy (D-RI) and Rep. Eric Cantor (R-VA) and in the Senate as S. 2042 by Sen. Debbie Stabenow (D-MI) and Sen. Johnny Isakson (R-GA) in August 2007 and September 2007, respectively. This legislation is supported by Families of SMA, the SMA Foundation, Fight SMA, and the Muscular Dystrophy Association. The passage of this legislation could change the lives of thousands of children and give them the future they so deserve….

As you know, legislation like this will only move through Congress with broad support and Members are significantly more likely to cosponsor and support legislation if their constituents are actively urging them to lobby for support of the bill on their behalf. Thus, to help move this legislation through the process WE NEED YOUR HELP IN SIGNING THIS PETITION to make sure your Senators and district Representatives know that this is an important piece of legislation to cosponsor.

To add your name to the petition, click here. For more information about the SMA Treatment Acceleration Act and other ways you can get involved, click here.

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There’s a new page on the Fight SMA website! The Spinal Muscular Atrophy Multimedia page will be home to videos, audio, photos, and other multimedia items about SMA and from events held by Fight SMA. You can go there right now to see the full version of Invisible Heroes, the video shown on the Fight SMA front page!

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 FB 16 [14:00m]: Play Now | Play in Popup | Download

This is the third in a series of Fighting Back Podcast episodes featuring audio from the Fighter Mom Friday event, held on May 9, 2008 in New York City.  The workshop was designed to educate, inform, and inspire attendees fighting against diseases or conditions affecting their children. The day-long event featured a number of speakers, and several of them have agreed to allow us to present in podcast form the information they offered during the event.

Episode 16 of Fighting Back features Dr. Alex MacKenzie, Director of the Children’s Hospital of Eastern Ontario Research Institute.  The audio for the episode was recorded during Dr. MacKenzie’s seminar.  He discusses the relationship between families and researchers, and how they can help each other.

More information on Fighter Mom Friday can be found on the Fighter Mom website.  More audio from the event is available on the Fighter Mom Multimedia page, or by subscribing to this podcast.

To listen to this episode, you can use the Flash mp3 player above, load the Fighting Back Podcast Feed into your favorite feed reader, subscribe via iTunes or other podcast directories, or simply use the download link above to load the file onto your computer.

If you have feedback, a comment or question, or are interested in having Fighting Back tell your story, please contact us via the Fight SMA contact page. You can also leave a message in the form of a comment on this blog as well.

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ABC News talked about a new SMA test earlier this month in an interview with Dr. Laurie Demmer from Tufts Medical Center.  The test is designed to be given to potential parents before pregnancy occurs.

To view the video in a popup, click here.

Note: An advertisement plays before the ABC News video starts.

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RICHMOND, June 6, 2008 — This week, the entire Virginia delegation to Congress has unanimously signed on as co-sponsors of the SMA Treatment Acceleration Act, a bill designed to increase federal support for research to develop a treatment for spinal muscular atrophy (SMA), the number-one inherited cause of infant death.

“This tremendous show of support and leadership will bring hope to thousands of families battling this deadly neuromuscular disorder,” said Martha Slay, president of Richmond-based FightSMA, one of the groups that is supporting the legislation.

“The Virginia delegation is setting a wonderful, bipartisan precedent for the rest of Congress and the Senate where we need to continue to build support this year for this legislation,” said Ms. Slay. “It is immensely gratifying that our home state has come through in such a big way.

“We particularly appreciate the support of Congressman Eric Cantor of Richmond, who was the original sponsor, along with Congressman Patrick Kennedy of Rhode Island. We would not be where we are today without Congressman Cantor taking the lead on this legislation,” said Ms. Slay.

The SMA Treatment Acceleration Act authorizes federal funding to:

• Upgrade and unify existing SMA clinical trials sites and establish a national clinical trials network for SMA.
• Establish a Data Coordinating Center to provide expert assistance and advice to SMA clinical trials sites.
• Expand and intensify federally supported research programs with respect to pre-clinical translational research related to SMA.
• Establish a research collaborative at the National Institutes of Health to ensure cooperation across multiple Institutes regarding research related to SMA.
• Enhance and provide ongoing support to the existing SMA patient registry in order to provide for expanded research on the epidemiology of SMA.
• Establish an SMA Coordinating Committee, consisting of representatives from relevant government agencies and the public, to coordinate government activities relating to SMA, serve as the principal advisor to agency heads, and conduct a study to identify barriers to the development of drugs for treating SMA and report findings and legislative recommendations to Congress.
• Require the Secretary of Health and Human Services to collaborate with the FDA and the Coordinating Committee to make recommendations for improving and expanding existing industry incentives to promote SMA drug development.
• Establish and implement a program for providing information and education on SMA to health professionals and the general public related to advances in the diagnosis and treatment of SMA and the provision of care to SMA patients.

The SMA Treatment Acceleration Act is supported by Families of SMA, FightSMA, the Muscular Dystrophy Association, and the SMA Foundation.

Co-sponsors from Virginia are: Senator John Warner, Senator Jim Webb, Congressman Eric Cantor, Congressman Rick Boucher, Congressman Jim Moran, Congresswoman Thelma Drake, Congressman Robert Wittman, Congressman Bobby Scott, Congressman Randy Forbes, Congressman Virgil Goode, Congressman Bob Goodlatte, Congressman Frank Wolf, and Congressman Tom Davis.

FightSMA, a nonprofit 501c3 corporation, was founded in Richmond, VA, in 1991 with the mission “to strategically accelerate research for a treatment and a cure for spinal muscular atrophy (SMA), the leading genetic cause of infant death.”

For more information, contact FightSMA at www.fightsma.org or 804.515.0080.

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 FB 15 [20:48m]: Play Now | Play in Popup | Download

This is the second in a series of Fighting Back Podcast episodes that includes the audio of seminars from Fighter Mom Friday. The workshop, held on May 9, 2008 in New York City, was designed to educate, inform, and inspire attendees fighting against diseases or conditions affecting their children. The day-long event featured a number of speakers, and a number of them have agreed to allow us to present in podcast form the information they offered during the event.

Episode 15 of Fighting Back Podcast features Louise Raymond, Senior Director of Global Corporate Responsibility for McGraw-Hill Companies. During her talk at Fighter Mom Friday, she discussed corporate giving and how to approach not only her companies, but other companies as well, to inquire about grants and funding for nonprofits.

Note: Ms. Raymond refers during her talk to a Powerpoint presentation. That presentation is available for download on the Fighter Mom Multimedia page.

Be sure to check back or subscribe to this podcast, so you don’t miss any of the upcoming episodes of Fighting Back!

To listen, you can use the Flash mp3 player above, load the Fighting Back Podcast Feed into your favorite feed reader, subscribe via iTunes or other podcast directories, or simply use the download link above to load the file onto your computer.

If you have feedback, a comment or question, or are interested in having Fighting Back tell your story, please contact us via the Fight SMA contact page. You can also leave a message in the form of a comment on this blog as well.

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A clinical trial entitled “Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I)” is currently recruiting type 1 SMA patients up to 9 months old. According to study description on ClinicalTrials.gov, investigators aim “to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.” The detailed description goes on to say:

Valproic acid (VPA) is a medicine that has been used for many years to treat patients with epilepsy. Recent research suggests that VPA may be able to upregulate expression of a backup copy of the SMN gene in SMA patient cell lines. In addition, some preliminary data suggests it may prolong survival in animal models of SMA. Because VPA can deplete carnitine in children with SMA Type I, carnitine is added to help prevent possible toxicity.

In this multi-center trial, we will evaluate the effects of VPA/carnitine on infants with SMA type I. A variety of outcome measures, including assessment of safety, will be performed at each study visit to follow the course of the disease. The protocol includes two baseline visits over a period of two weeks, two clinical assessments on medication at 3 and 6 months, and then 6 months additional followup via telephone. Total duration of the study will be approximately 12 months.

This is an international, multi-center trial with study sites at:

• Johns Hopkins University in Baltimore Maryland
• Children’s Hospital of Michigan in Detroit
• Ohio State University Medical Center in Columbus
• University of Utah/Primary Children’s Medical Center in Salt Lake City
• University of Wisconsin Children’s Hospital in Madison
• Hospital Sainte-Justine in Montreal, Quebec, Canada
• Klinikum der Universität zu Köln in Cologne, Germany

For more details about the clinical trial, including eligibility criteria and contact information, click here. For more information about clinical trials in general, click here.

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A quick update on the fallout from PerezHilton.com posting about Fight SMA on Monday, May 26:

As we told you in this blog post, the folks at PerezHilton.com recently began posting information about what they call “worthwhile causes” on their site every week or so. On Monday, Perez included Fight SMA in that list. The site posted a link to fightsma.org, a description of our organization and spinal muscular atrophy, and showed a video that we posted on YouTube about SMA sufferer Billy Therriault and his family.

Since that posting on PerezHilton.com, nearly 30,000 people have viewed the Billy Therriault video. It’s gone positively viral, and we’re thrilled about it! Additionally, thousands of new visitors have found the Fight SMA website and learned about this horrible disease.

Here’s just a sampling of the dozens and dozens of comments that have been logged on the PerezHilton site:

Smile says –
I was shocked to see sma awareness on this site. I think it’s great.  My niece died from this at the age of two. It’s an awful disease!!!! Thanks Perez!
—————————————-
Kelly says –
Perez,
Thank-you for posting this wonderful video about our very dear friend Billy. My son will be 3 in June and also has Type 1 SMA and we live in the same town as Billy. These kids are the TRUE news out there. This IS very important and this disease does NOT discriminate. ANY celebrity…any PERSON reading this website could be a carrier of the SMA gene. While my child and EVERY SMA child is a wonderful blessing…this is a hard life and for your readers who don’t think it is something they should pay attention to, they are very wrong. The more information about SMA the better and the sooner we find a cure the better. Bravo for posting about this disease that affects my child and many others out there.
—————————————-
Maggie says –
My eyes teared up watching that Video. I have two little boys of my own, and I feel blessed that they are healthy. To think that 1 out of every 40 people in the us carry the gene for sma is scary to think about. My heart goes out to the kids and families of the kids that suffer from sma….. I am going to donate we need to find a treatment.

If you haven’t seen the Billy Therriault video, you can view it on the front page of Fight SMA, or on YouTube. See the video everyone is talking about!!! If you haven’t seen the Perez Hilton post, you can find it here.

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Columbia University TV ran a great story during their student-run newscast about Fighter Mom Friday.  The Fighter Mom Friday event was on May 9, 2008 in New York City.  It was a day-long workshop for mothers (and others) fighting against serious or life threatening conditions affecting their children.

You can view the Columbia University TV newscast by clicking here.  The FighterMom Friday story is the last one in the newscast.  To save time, you can fast forward.

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We’re thrilled to announce that one of the most popular celebrity gossip blogs on the web today chose Fight SMA as one of its “worthwhile causes”. Recently PerezHilton.com began posting information about different charity sites each week. Taking the suggestion of Fight SMA Public Affairs Coordinator Caroline Gibson, the site included Fight SMA in its list. With that listing came a mention on the front page of the wildly popular site, a link back to FightSMA.org, and a blog post that included our most recent YouTube video (also seen on the front page of FightSMA.org).

Click here to read the post on PerezHilton.com!

We’re grateful to PerezHilton.com for telling more people about SMA and for the publicity for Fight SMA. Thank you!

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