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:

Less 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.

Following the publication of his team’s gene therapy work with AAV-9 in spinal muscular atrophy (SMA) mouse models (Foust et al., Nature Biotechnology), Dr. Brian Kaspar participated in Nationwide Children’s Hospital’s monthly podcast about neuromuscular research. In this podcast, Dr. Kaspar spoke about a number of topics including:

• What is spinal muscular atrophy (SMA)
• The genetics of SMA
• The difficulty developing treatments for SMA
• The results AAV-9 has shown in SMA mouse models
• The need for additional studies before moving to human trials
• The next steps toward the clinic

The podcast is available by clicking here and scrolling to the May podcast. At the time of this post, there is a problem in how the podcast is available on the Nationwide Children’s Hospital website. The audio file has a comma added at the end of the file type which prevents players from recognizing file type. In order to play the podcast, simply download it to your computer, select the file name, and remove the comma. Your computer should then recognize the podcast as a MP3 file.

Recently, FightSMA launched the “Realizing the Dream” fundraising campaign which partners with the Gwendolyn Strong Foundation to advance a common goal: to bring SMA gene therapy to clinical trial. These efforts will support Dr. Kaspar and other collaborating scientists. To learn more about the gene therapy campaign and how you can get involved, click here.

Gwendolyn Strong Foundation Is Campaign Partner

RICHMOND, VA - Richmond-based FightSMA announced today a new fundraising campaign for spinal muscular atrophy (SMA) gene therapy, “Realizing the Dream.” FightSMA is collaborating with the Santa Barbara–based Gwendolyn Strong Foundation, to form a bi-coastal partnership with a common goal: to bring SMA gene therapy to clinical trial.

“Ten years ago, it would have been unheard of to say scientists were approaching a treatment or cure for spinal muscular atrophy,” said FightSMA President Martha Slay. “Today, the dream is being realized in some of the most prestigious labs across the country. Never before has there been such promise for SMA gene therapy.”

Beginning now and for the next three years, families and groups in the SMA community will raise funds to build a safe foundation, deliver genes to an SMA model, and produce adequate vector (gene delivery) supply. These efforts will support Dr. Brian Kaspar (pictured left) of Nationwide Children’s Hospital and The Ohio State University and other collaborating scientists.

FightSMA, working with the Gwendolyn Strong Foundation, a funding source for critical SMA science and awareness initiatives, invites the SMA community to make a decade-old dream come true. “Our SMA gene therapy program at Nationwide Children’s Hospital and the Ohio State University continues to show great promise for treating SMA patients,” said Dr. Kaspar.

FightSMA’s objective for the balance of 2010 is to complete funding for Phase One and for Year-One of Phase Two of the research program. “The first objective is to build a solid foundation of safety and to eliminate toxicity,” said Dr. Chris Lorson, FightSMA Science Director. Additionally, FightSMA plans to raise another $250,000 to fund the first year of Phase Two (Delivery & Efficacy). The “Realizing the Dream” program will be accomplished through a series of campaigns. Completing these two Phases will bring SMA gene therapy significantly closer to clinical trial.

FightSMA has been instrumental in helping to develop a gene therapy strategy to cure spinal muscular atrophy (SMA), including oligonucleotides and gene replacement vectors. The strides that SMA researchers have made in the gene therapy arena have provided insights into a range of genetic disorders, including other neurodegenerative disease (ALS/Lou Gehrig’s disease, myotonic dystrophy, Huntington disease) and other diseases such as Duchenne muscular dystrophy.

For more information on the FightSMA-Gwendolyn Strong Foundation partnership and “Realizing the Dream” campaign, visit www.fighsma.org or call 804-515-0080.

FightSMA was created to strategically accelerate the search for a treatment and cure for spinal muscular atrophy (SMA), the number-one inherited cause of infant death. The organization pursues this objective by raising awareness and funding for SMA research.

Every June, daughters, sons, mommies, sisters, brothers, and friends head to their local drug store to pick out the perfect card for someone they know that’s a terrific father. Some cards will make him laugh; others might make him cry (though he’d never show it). He’ll rest the card on the desk in his office, a smile crossing his face each time he recalls the kind, handwritten words written inside, punctuated with a “Happy Father’s Day.”

He’ll receive electronic gadgets, tools, ties, and socks. He might even get breakfast in bed. But the greatest gift of all that day is the reason why we celebrate him in the first place: his CHILD.

They say a parent’s love is the most powerful love in the world. You all know that. The love you feel for your children is deep and unconditional, ever growing AND ever present. You would go to the ends of the Earth for your child, and beyond if you could. And today, Dad, we honor you. We thank you.

The SMA community is overflowing with love for our precious children. The level of understanding between the parents runs deep. This is a special community, filled with special parents, and most importantly, special kids.

FightSMA invited a few special dads to share special Father’s Day story with each of you:

Mike Calise, Dad to Corinna
Cranston, RI
Corinna’s Angels (www.corinnasangels.org)

• What has being a Dad meant to you?
  Being a dad is a tremendous joy and responsibility. Your priorities change the second you have a child; you and your spouse share an overwhelming sense of love, gratitude and responsibility as a result of having a child together. Being a dad of a child with a disability is even more challenging but the rewards are so much greater. There is no greater feeling than sharing special times with my daughter, Corinna. I really appreciate every bit of fun that we have and every new experience we are able to share.
• What is your favorite thing to do with Corinna?
  I love to just hang out with Corinna and play the Wii or Nintendo or just watch her favorite movie or TV show. Corinna has an incredible spirit and great sense of humor that makes every minute with her enjoyable.
• Do you have a special message for the other dads this Father’s Day?
  Words of wisdom to other fathers: Appreciate and love your child and family like it could be your last day together. At the end of the day, there is no greater feeling of love than that which you share with your family.

Bill Strong, Dad to Gwendolyn
Santa Barbara, CA
Gwendolyn Strong Foundation (www.gwendolynstrongfoundation.org)

• What has being a Dad meant to you?
  Being Gwendolyn’s Dad means the world to me. Regardless of how short my time with her will be, I consider myself unbelievably fortunate to have been given the gift of time with my precious daughter. Over the past 2 1/2 years, I’ve learned more from Gwendolyn about myself and what is truly important in life than I had over the previous 31. Getting to meet Gwendolyn has changed my life, challenging myself to truly know her and always be present with her continues to fulfill me, and striving to provide her the quality of life she deserves and cherishing every second that I have left with her is and always will be my number one priority.
• What’s your favorite thing to do with Gwendolyn?
  My favorite things to do with Gwendolyn are anything and everything that sets off that soft, infectious giggle, inspires that sparkle in her eye, and strengthens that deep connection between the two of us. I love my “Daddy Saturday” walks together, finding new crazy adventures, reading her books, teaching her new things, our snuggle time, and being the last one she sees at night and the first one she sees when she wakes up. More than anything, I simply love spending every possible second — focused on the present — together with Gwendolyn.
• Do you have a special message for the other dads this Father’s Day?
  Happy Father’s Day to all of the dads who are fortunate enough to have an incredible daughter/son in their life like Gwendolyn.

Today, Friday, January 22nd, is the last day to cast your vote in support of Spinal Muscular Atrophy research in the Chase Community Giving campaign on Facebook.

The charity that receives the most votes will win the top prize of $1 Million and five runners-up will get $100,000 each. The Gwendolyn Strong Foundation (GSF) is trying hard to hold on to 6th place - a $100,000 eligible position.

GSF has pledge to give 100% to SMA awareness and research deemed most promising by the SMA community. Within 90 days after winning the Chase Community Giving prize, GSF will execute a unique online voting campaign and distribute all funds to the winning programs recommended by prominent and committed SMA scientists and voted most important by the SMA community. Thanks to the first round of voting, the Gwendolyn Strong Foundation (GSF) won $25,000 which has already been distributed to research.

You can only vote for the GSF once, so in order for SMA to have a chance at this critical funding, we need you to not only vote on Facebook, but get your friends and family involved as well.

Go to http://VoteForSMA.com to vote and to learn more.

Thanks to previous votes, the Gwendolyn Strong Foundation (GSF) won $25,000 for Spinal Muscular Atrophy (SMA) research in the Chase Community Giving campaign on Facebook. Now, for only one week from January 15th to January 22nd, SMA has an opportunity to win much, much more!

The charity that receives the most votes will win the top prize of $1 Million and five runners-up will get $100,000 each. GSF has pledge to give 100% to SMA awareness and research deemed most promising by the SMA community. Within 90 days after winning the Chase Community Giving prize, GSF will execute a unique online voting campaign and distribute all funds to the winning programs recommended by prominent and committed SMA scientists and voted most important by the SMA community. You can only vote for the Gwendolyn Strong Foundation once, so in order for SMA to have a chance at this critical funding, we need you to not only vote on Facebook, but get your friends and family involved as well. Go to http://VoteForSMA.com to vote and to learn more.

Congratulations to our friends at the Gwendolyn Strong Foundation. Their phenomenal outreach across the internet successfully garnered enough votes for the Foundation to be named to Top 100 list in the Chase Community Giving Campaign on Facebook. This honor comes will a $25,000 award and the chance to move on to round two where they will compete for a $1 million grant.

From the Gwendolyn Strong Foundation:

December 16, 2009
Gwendolyn Strong Foundation Top 100 Charity, Wins $25,000 Grant From Chase Facebook Campaign

This is truly unbelievable, incredible!

Today, we received news from Chase that the Gwendolyn Strong Foundation was selected as one of the top 100 charities, based on YOUR votes, in their Chase Community Giving campaign on Facebook. Thus, GSF will receive a $25,000 grant from Chase and will advance to round 2 of the campaign with a chance to win as much as $1 Million.

We’re still gathering information and to be honest it’s still sinking in, but we wanted you all to know this amazing news right away since none of this would have been possible without each and every single one of your votes and your personal, passionate efforts to prop the Gwendolyn Strong Foundation up on your shoulders. This is another humbling example of how this vibrant, active SMA community and every person that this horrible disease touches can collectively accomplish anything that we put our minds to as one, united, motivated voice.

We are excited to be donating 100% of this grant to Dr. Hans Keirstead’s promising SMA stem cell program at UC Irvine. As most of you know, this groundbreaking research has the potential to cure SMA and have a material, positive impact on all science forever. We know that this grant will help Dr. Keirstead and his team accelerate the process to get this treatment to human clinical trials as safely and efficiently as possible and to the children in need of a cure.

From the bottom of our hearts, thank you, thank you, thank you to each and every one of you. Now, on to ROUND 2! As a community, let’s go get that $1 MILLION to help CURE SMA once and for all!

Click here to go to the Chase Community Giving campaign on Facebook to see the top 100 charities.

Click here to read the press release from Chase.

Don’t know what spinal muscular atrophy (SMA) is? Learn more here.

This request comes from Bill and Victoria Strong of the Gwendolyn Strong Foundation and Neda Zadeh, M.D., Medical Genetics Fellow at Stanford University. They are reaching out to mothers of children with spinal muscular atrophy to ask if they would participate in a study that “may help pave the way for SMA carrier screening to be offered to more women.”

From: Neda Zadeh, M.D. — To the Claire Altman Heine Foundation:

You are invited to participate in a research study on the possible association between Nuchal Translucency (NT) measurement and fetuses affected with Spinal Muscular Atrophy (SMA). Our goal is to determine whether there is an association between increased NT measurements and SMA. If so, diagnostic testing for SMA may be offered to women with increased NT and no evidence of a chromosome abnormality of the fetus. We are only recruiting mothers of children confirmed to have SMA by molecular testing.

Involvement in this study is entirely voluntary and confidential. It will require your permission to access particular medical records for both you and your child. Your participation will not involve invasive procedures such as blood draw or tissue sampling. There will be no monetary compensation for your participation.

If you are interested in participating, or would like to hear more about this study, please contact me at (650)721-1439.

Sincerely

Neda Zadeh, M.D.
Medical Genetics Fellow
Stanford University
Division of Medical Genetics

Last week, Bill and Victoria Strong shared a story of meeting a man, Joe Barnick, who has inspired them in their care for their daughter Gwendolyn, who, like Joe, has spinal muscular atrophy (SMA).

From the Strong’s blog:

November 20, 2009

Joe Barnick, thanks for the inspiration!

This is such a small, small, tiny world in so many ways. Victoria and I have been researching and thinking through ways to use technology to help Gwendolyn communicate. There is a really cool application that we’ve been looking at, Proloquo2Go, that brings augmentative and alternative communication (AAC) to the iPhone/iTouch devices. It looks like it could potentially help Gwendolyn. I emailed support at AssistiveWare, the company that created the application, to ask a few questions and I received a very thorough response from a gentleman named Joe Barnick. It turns out that not only is Joe a very helpful customer support person at AssistiveWare, but Joe also has SMA. He is also the designer and editor-in-chief of the AssistiveWare Newsletter. He was very helpful and he sent me the YouTube video below that shows how he uses his Mac and an AssistiveWare application called KeyStrokes for “all the things he cannot do in the real world”. I feel so fortunate to have met Joe — for so many reasons. He’s an inspiration to me and he reminds me to never underestimate Gwendolyn’s abilities and — in his words — to “never stop fighting for your daughter and never give up hope! : )”. Joe, I can promise you that I never, ever, ever will!

I’m so glad that I met you Joe. Thank you for opening up my mind even more to what is possible and I look forward to keeping in touch! Keep up the great work at AssistiveWare…

Bill Strong, who founded the Gwendolyn Strong Foundation with his wife Victoria and who recently joined the FightSMA Board of Directors, was invited to do a radio interview for “Focus on Issues.” This wonderful interview, which aired four times during this past weekend, covers a broad range of subjects from how the Strong’s daughter Gwendolyn is doing to the need for more research to how individuals can help in the fight against spinal muscular atrophy (SMA).

Click here to listen to the whole interview