The below video has been posted on this blog before, but we noticed the other day that it’s had quite a number of views and comments on YouTube. That’s why we thought we would post it on the Spinal Muscular Atrophy Blog again, for those who haven’t yet seen it. It’s a fabulous video, created by Jim Vaile. It was shown in April, at the 2007 Fight SMA Spinal Muscular Atrophy Conference in Washington, DC. It features images of the children of Fight SMA, the reason for our fight. Watch the video and, if you can, post a comment by clicking on the video and visiting the YouTube site. The more “views” counted by YouTube, the higher the chances that the video will maintain a prominent position.

Enjoy!

Below is a new article written for Fight SMA and FighterMom. Some material in this article comes from the FighterMom Manual.

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You CAN Make Things Happen in Washington for Your Cause

Fight SMA, the organization my husband and I founded in 1991, is an international nonprofit organization working toward a cure for spinal muscular atrophy (SMA). We haven’t always been a big organization, however. As with all grassroots efforts, we started small and had a lot to learn.

When we launched Fight SMA we knew we had to work hard to get the federal government to increase funding for spinal muscular atrophy. We met with our congressional representatives and with the head of the relevant institute within the massive National Institutes of Health. We received useful insights and nice photographs for our newsletters, but failed to increase the small NIH budget for spinal muscular atrophy research.

Our fortunes changed, however, as we gained experience. Our latest successes involve the SMA Treatment Acceleration Act, proposed legislation that was recently introduced for consideration in the U.S. House and Senate. The bill was created by a coalition of SMA groups that includes the organizations Families of SMA, the SMA Foundation, and Fight SMA.

Below is a list of the some of the things we’ve learned. Donna Mullins of Washington, D.C. public affairs group Winning Strategies and Steve Eichenauer of Public Strategies Washington provided these tips. Hopefully they will help you have more success the FIRST time you solicit the federal government for your cause.

The Power of Partnerships

Our first successes with Congress came when we “threw in” with other SMA organizations that had taken on Washington as a strategic priority. As part of a coalition, we were harder to ignore, and thus started seeing real results. Research what other organizations are already doing to raise funds or awareness for your cause and, if possible, partner with them. Be sure, however, that your goals are one in the same.

Additionally, when scheduling appointments with a representative, it’s best to book a group of visitors. A “pack” of constituents may be more likely to get face-to-face time with a representative.

How to “Attack the Government” from Multiple Fronts

While it’s an excellent idea to travel to Washington, you can also bring your representative to you. Contact the home district office and invite him or her to an event you’re sponsoring. Engagement at home, when they’re working to impress voters, can be a great way to get their attention.

Know What You Want

This can be one of the hardest things to figure out. Some representatives are willing to take up the cause of raising awareness and work toward more funding for a cause. Others believe that the scientific community should drive funding decisions. Additionally, many elected representatives fear that taking up one cause will open themselves up to criticism from constituents who are pushing other causes. As a guide, find out what they have done on behalf of others. Make it a goal to get at least as much for your cause.

What To Avoid

It is important to know what your congressional representative can or cannot do. For example, members of Congress cannot dictate where the NIH spends money. However, they can draw attention to the concerns of a constituent and raise questions about the level of funding.

Make the Most of Your Time

When you sit down for your “Big Washington Meeting”, tell your representative what you think the government SHOULD be doing, and ask directly for their assistance. This is not the time to be bashful.

Don’t be surprised if your representative doesn’t show up for your meeting, and don’t underestimate the role of the staffer who is there. That person may be your regular point of contact, so build a relationship with them.

Cut to the Chase

Finally, when you do sit down with your elected representative or his/her staffer, start talking right away about what you want. Legislators and their staff are very busy, so this is not a time to be long winded. When you present your case, be sure to relate it back to his or her home district. Some statistics about how your cause affects their home state or district can be extremely effective.

Bring Materials

Create a one-page document to give your representative or his/her staffer during your visit. The document should be bulleted and easy-to-read, and include all of the important information about your cause, including contact information for you or your organization.

Don’t be Afraid to Follow Up

On any given day in Washington, members of Congress have about 20 different people visiting with them and seeking assistance. After your meeting, be persistent. Phone or write to remind your representative what was discussed and what was promised.

While following this plan doesn’t guarantee success, not having a plan will nearly guarantee failure. Good luck in your efforts.

To learn more about the SMA Treatment Acceleration Act, visit the Fight SMA website. You can also listen to episode three of Fighting Back Podcast, a program produced by Fight SMA and FighterMom, to hear Martha Slay discuss recent Fight SMA congressional visits.

The outpouring of support for Callum Robbins, son of musician and founder of the band Jawbox, J. Robbins, continues to flow.  Callum, who will be two years old in January, has Spinal Muscular Atrophy Type 1.  This comes this week from music magazine Harp:

This December, cellist Gordon Withers of Boston indie group Betwixt will also add to the cause with Jawbox on Cello: A Benefit for Cal Robbins, a collection of 12 Jawbox songs performed by Withers. The album will be released digitally through Ioda on Dec. 18, is also available on Withers’MySpace page and can be streamed on his Virb page. All proceeds will benefit the Cal Robbins Care Fund.

Read the Harp article here.  To read more on Callum, visit the blog J. Robbins set up for him.

There’s an article in the latest issue of Neurology Today that covers the recent SMA Drug Summit. The summit, held in Washington D.C. and sponsored by Fight SMA, Families of SMA, and SMA Foundation, was held to foster the development of spinal muscular atrophy drugs that could be tested in clinical trials facilitated by the International Coordinating Committee.

The article includes an update on some of the drugs currently being tested that might show promise in treating SMA.

Click here to read the story, in PDF format.

There’s a wonderfully written story of a family’s life with spinal muscular atrophy (SMA) published this week in The Olympian newspaper from Olympia, Washington.  The article, written by Diane Huber, tells the story of the Lormers and their three year old daughter Kayleigh.  Kayleigh wasn’t expected to live past her second birthday, but this week she’s starting preschool.  Ms. Huber describes the little girl as a “fighter”.

She loves watching “Dora the Explorer,” coloring and reading books with her parents, Cindy and Dean Lormer. She has to be reminded to say please and thank you. Her favorite colors are purple, pink and red.

But she’s trapped in a body that’s dying.

Kayleigh has spinal muscular atrophy, a genetic disorder that attacks nerve cells in the spinal cord. The disease has made Kayleigh’s muscles so weak that she can’t crawl or walk or hold up her own head.

Yet she is a fighter.

Doctors predicted Kayleigh wouldn’t live past age 2, but on Oct. 29, she celebrated her third birthday. She got a new pink polka-dotted dress and a V-Smile, an educational video game console.

And this week, she’ll start preschool.

The Lormors want to provide their daughter with the best quality of life they can. She has taught them about life and how much they can bear.

“It changed everybody in the family. It made you really take a step back and appreciate what you have,” Cindy Lormor said.

They are all too aware that each day with their daughter is a miracle.

Visit the website for The Olympian to read the entire story online

14-year-old Megan Owen of Lancashire, England, is getting a treat that many adult women would give their eye teeth to receive. Megan fascinated with fashion and pretty shoes, the same as many other girls her age. Unfortunately, Megan has Spinal Muscular Atrophy (SMA), making most shoes unavailable to her. Here’s more from the Lancashire Evening Post:

Over the years, Megan’s disease has got progressively worse and she can now no longer move any of her limbs at all.

Megan’s condition means her feet point downwards like a ballet dancer’s and she cannot bend them.

As a result, she cannot wear shoes, although she is fascinated by them and longs to own a pair.

Her mum Karen said: “Megan is a real girly girl and loves pink and glitter and is obsessed with shoes.

“When Megan was born, we thought she was a normal healthy little girl.

The Make A Wish people are making sure Megan gets some special treatment related to her love of footwear. Famed shoe designer Jimmy Choo has promised to close his exclusive London store in order to give Megan a personal tour.

Make A Wish grants wishes for children with life-threatening conditions.

For more about Megan’s wish, read the story in this week’s Lancashire Evening Post.

It turns out YouTube can be of more use than looking for silly videos.  Brad Fisher’s daughter, Shira, has Spinal Muscular Atrophy Type 1.  Shira is now two years old, and over the past few months Brad has posted several home grown how-to videos related to the things parents need to do to take care of their SMA kids.  The videos run through the steps needed to perform a cough assist and change a “mickey button”.  While Mr. Fisher isn’t a doctor, and you should always consult your doctor with questions about procedures, it’s extremely helpful to SMA parents to see how others perform these tasks.

Click here to view all of Shira’s videos on YouTube.

To learn more about Shira, visit her website.

A new article from researchers at Peninsula Medical School in Exeter, UK charts out ongoing studies at the school that they hope will lead to a treatment for some victims of spinal muscular atrophy (SMA).

Researchers […] have carried out studies into one of the two survival motor neuron (SMN) genes that set suffers from SMA apart from the rest of the population. In 98% of SMA sufferers, the SMN protein is only produced from one of these genes. The resulting fall in SMN protein levels is what causes SMA to develop.

The remaining two per cent of SMA sufferers produce normal levels of SMN protein, but the protein is altered so that it is no longer capable of performing its normal functions within the body. One of the main reasons for this is that these disease-causing alterations in the SMN protein prevent it from localising to the correct places in the body¡¯s cells. By looking at why mutations occur and what they do to divert protein away from the cellular areas where it is required, the Peninsula Medical School team may be able to identify compounds that alter the protein so that it is directed correctly ¨C effectively producing a treatment for the disease.

The research could also have potential bearings on other treatments, such as gene therapy.

The paper highlighting this research is the first to be published by this particular research team, and it has made the front cover of Human Molecular Genetics, a remarkable achievement for a first time publication.

Read the entire article on EurekaAltert. 

FightSMA New York’s Second Annual Gallery Walking Tour will take place on Saturday, Nov. 3 from 2-4 pm at some of Chelsea’s most innovative and inspiring galleries.

New York, New York (PRWEB) October 31, 2007 — Participants in an upcoming walking tour of art galleries in New York’s Chelsea neighborhood will not only learn about featured artists and the inner workings of art galleries, they’ll also raise money for a child-killing disease. The SMA Walking Tour, to be held on November 3, 2007, will raise funds for research into spinal muscular atrophy. The tour will visit six different locations over a two hour period, allowing participants a behind-the-scenes look at how art galleries operate.

Spinal muscular atrophy (SMA) is the leading genetic killer of children under two. The neuromuscular disease causes a wasting of the muscles, robbing its victims of the ability to run, walk, crawl, play and, in many cases, even breathe or swallow.

“I’d like to thank the participants of the walking tour in advance for their support,” said Kathy Tompkins, chair of FightSMA’s New York chapter. “It’s critical that we find a cure for this disease, particularly since research into SMA benefits dozens of other disorders.”

The event is organized by Andrew’s Buddies of New York, the New York chapter of international nonprofit Fight SMA. The tour will begin at 2:00 p.m. and end at 4:00 p.m. Participants will start at Rare Gallery (521 West 26th Street) and continue on to DAmelio Terras, Friedrich Petzel, Nicole Klagsbrun, Lombard-Freid Projects, and Postmasters Gallery. Each gallery director will speak about the style of the emerging artist featured in the current exhibition.

The suggested contribution is $25 per person.

For more information ont the walking tour, please visit www.fightsma.org or call Caroline Gibson at (804) 515-0080.

About Fight SMA:
Headquartered in Richmond, Virginia, Fight SMA (also known as Andrew’s Buddies) is an international nonprofit group dedicated to accelerating research for a treatment or a cure for spinal muscular atrophy (SMA), a neuromuscular disorder that kills more babies than any other genetic disease. For more information on spinal muscular atrophy and Fight SMA, please visit http://www.fightsma.org The latest SMA news and research information is available at the Spinal Muscular Atrophy Blog, at http://www.fightsma.org/blog