Gwendolyn Strong Foundation Is Campaign Partner

RICHMOND, VA – Richmond-based Fight SMA announced today a new fundraising campaign for 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 (SMA),” 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 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.

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(Richmond, Virginia ) —Richmond, Virginia-based FightSMA, a nonprofit dedicated to developing a treatment for the deadly childhood crippler spinal muscular atrophy (SMA), is awarding a $250,000 research grant to Dr. Brian Kaspar, a principal investigator at Nationwide Children’s Hospital and associate professor of The Ohio State University.

The grant will support safety and toxicity studies for AAV(-9), a potential “delivery system” for gene therapy for the treatment of SMA. “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. “With this award, we will be able to test our therapy for potential safety or toxicity issues and provide important information for the regulatory process as we advance our program to the clinic in a swift and safe manner. We are all very thankful for the support of FightSMA.”

The grant has been made possible by funding both from FightSMA and from its associated partner organization, The Gwendolyn Strong Foundation. “We are tremendously excited about the potential for gene therapy in SMA and our continuing collaboration with The Gwendolyn Strong Foundation and the SMA Community,” said FightSMA President Martha Slay. “Gene therapy research is a marathon run, but the work of Dr. Kaspar will advance the field significantly for SMA.”

Spinal muscular atrophy (SMA) is the leading genetic cause of infant death. SMA is caused by the absence of a gene that is critical for muscle strength, the SMN (“survivor motor neuron”) gene. That gene is absent in approximately one in 30 individuals. If both a man and woman are lacking the necessary SMN gene, the chances are 25% that any of their children will manifest SMA.

An objective of gene therapy is the restoration of the missing gene through delivery systems called “vectors.” Adeno-associated virus (AAV) is considered to be an attractive candidate for the construction of vectors.

FightSMA was created in 1991 with the mission to “strategically accelerate the development of a treatment or a cure for spinal muscular atrophy.” Working with the guidance of an international Scientific Advisory Committee, FightSMA has awarded SMA research grants at more than 40 universities and research institutions in the United States, Canada, the United Kingdom, France, and Italy. FightSMA has chapters across the United States and in Canada.

For more information, contact: www.fightsma.org or 804.515.0080

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This release also available at:
PRWeb: FightSMA Awards $250,000 Grant to Gene Researcher at The Ohio State University
PRLog: Fight SMA Awards $250K Grant to Researcher at OSU

FightSMA Canada has been awarded a C$250,000 research grant from CML HealthCare of Mississauga. The Children’s Hospital of Eastern Ontario (CHEO) Foundation will match the grant, providing a total of C$500,000 to advance spinal muscular atrophy (SMA) research. The multi-year grant will support investigation into “clinic-ready agents” that can increase the presence of Survivor Motor Neuron, a protein that is essential for muscle strength and is deficient in children diagnosed with SMA.

“SMA is a devastating disease,” said CHEO research institute’s Dr. Alex MacKenzie. “It is the number-one genetic cause of death among infants.  This generous gift from CML HealthCare and the CHEO foundation will be key to moving the fight against SMA forward and will provide the thousands of children fighting this terrifying disease with hope for an effective treatment in the future,” said Dr. MacKenzie.

“CML HealthCare is excited about supporting the fight against SMA,” said Kent Nicholson, Executive Vice President and Chief Operating Officer. “We are very impressed and inspired by the determination and dedication of the researchers and families fighting SMA, and we are proud to be a part of the fight.”

“I want to express my appreciation for the support that CML HealthCare has provided and for their continued dedication in helping us find a cure for SMA,” said Tracy Lacey, President of FightSMA Canada. “CML’s efforts have made our recent research funding campaign one of the most successful that we have ever had.”

As a leading Canadian provider of diagnostic medical services, CML HealthCare is dedicated to providing patients with Care. Confidence. Comfort.TM The nearly 40-year-old company is one of largest providers of medical imaging services in North America and one of the largest community-based providers of laboratory testing services in Ontario.

“It is companies with vision, like CML HealthCare, that are truly changing our world,” said Martha Slay, President of FightSMA. “We all benefit from their generosity and from their example.”

FightSMA is an international nonprofit corporation dedicated to developing a treatment for spinal muscular atrophy (SMA). FightSMA has awarded SMA research grants to more than 40 universities and research institutions in Canada, the United States, the United Kingdom, France, and Italy.

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CML HealthCare of Mississauga, Ontario has partnered with FightSMA Canada, to help find a cure for spinal muscular atrophy (SMA), the leading genetic cause of death among children under the age of two. The awareness of the devastating impacts of SMA is low among the general public. The new partnership will provide a greater voice in the efforts to raise funds and increase awareness of SMA in communities across Canada.

Spinal Muscular Atrophy (SMA) refers to a group of diseases, which affects the motor neurons of the spinal cord and brain stem. The disease does not discriminate on the basis of gender or ethnicity.

“CML HealthCare is proud to be a partner in the fight against SMA, “said Paul Bristow, President and Chief Executive Officer. “We take great pride in supporting research that gives those individuals with SMA a fighting chance.”

“FightSMA is excited to formalize our partnership with CML Healthcare in our efforts to find a cure. Working with CML over the past 2 years we have done great things together and we look forward to the future opportunities that this partnership provides to raise money and awareness for all of the individuals and families affected by SMA” Said Tracy Lacey, President of FightSMA Canada.”

As a leading Canadian provider of diagnostic medical services, CML HealthCare is dedicated to providing patients with Care. Confidence. Comfort.^TM The nearly 40-year-old company is one of largest providers of medical imaging services in North America and one of the largest community-based providers of laboratory testing services in Ontario.

FightSMA is an international nonprofit corporation dedicated to developing a treatment for spinal muscular atrophy (SMA). FightSMA has awarded SMA research grants to more than 40 universities and research institutions in Canada, the United States, the United Kingdom, France, and Italy.

(Santa Barbara, CA) – Millions of people ‘Tweet’ every day, and Bill Strong knew there had to be a way to harness all of that online talk to help end spinal muscular atrophy, a disease that is affecting him very personally. With a Twitter application called ‘Tweet for a Cure’ and the help of thousands of Twitter users, he’s making progress in his quest to find a cure.

Bill and Victoria Strong’s daughter, Gwendolyn (seen to the right), was diagnosed with spinal muscular atrophy (SMA) at the age of six months.  SMA is a neuromuscular disease that causes weakness and wasting of the muscles.  In the most severe cases, it can rob its victims of the ability to crawl, sit, walk, breathe, eat, talk, or even smile.  SMA is the leading genetic killer of children under two.

Not long after the SMA diagnosis, Bill and Victoria founded the Gwendolyn Strong Foundation to raise awareness of and money for SMA research.  Bill also serves on the board of directors of Fight SMA, a larger organization with a mission of strategically advancing research for SMA.

Bill has harnessed the power of the Internet and social media for several campaigns that further his cause of raising much needed awareness of SMA.  His latest effort is “Tweet for a Cure”.  The Twitter application helps users send a “tweet” to their local legislators.  The message asks the legislators to support the SMA Treatment Acceleration Act.  The proposed legislation in the United States, written and backed by multiple SMA organizations, would help to clear the way for researchers to find a cure for the disease.  Within one month of its launch, more than 2,600 people utilized “Tweet for a Cure”, reaching nearly two million Twitter users.

“Using social media, such as Twitter, Facebook, and even online petitions, organizations like ours can reach large numbers of people with less effort than it took just a few years ago,” said Strong.  “We’re grateful for the individuals who have used ‘Tweet for a Cure’ and for those who have signed our online petition, and hope that they continue to support our efforts to help end spinal muscular atrophy.“

Bill’s online petition, which asks that legislators support the SMA Treatment Acceleration Act, can be found at http://www.petitiontocuresma.com.  Since being created in July of 2008, it has gathered approximately 70-thousand digital signatures.

To find the “Tweet for a Cure” application, go to http://www.endsma.org/twitter.  To learn about the Gwendolyn Strong Foundation, go to http://www.endsma.org.  For more about spinal muscular atrophy, go to Fight SMA’s website, at http://www.fightsma.org.

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. The latest SMA news and research information is available at Fight SMA’s Spinal Muscular Atrophy Blog, at http://www.fightsma/blog.

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This release also found at:
Online PR News: Thousands Voice Support for Spinal Muscular Atrophy Research with “Tweet for a Cure”
PRLog: New Application Helps Twitter Users Contact Legislators about Spinal Muscular Atrophy

Richmond, VA (June 16, 2009) – The Fight SMA 2009 Annual Spinal Muscular Atrophy Conference, The Good Fight, featured many firsts.  One of the most important to SMA families was the first-ever webcast of the “Friends and Family” portion of the conference.  Now, Fight SMA is announcing that the video from that webcast is available for online viewing and download.

Founded in 1991 in Richmond, Virginia, Fight SMA is an international non-profit organization dedicated to finding a treatment or cure for Spinal Muscular Atrophy (SMA), the leading inherited killer of children under two.  The neuromuscular disease affects the victim’s motor neurons, causing muscle atrophy, loss of strength, and disability.  The majority of children who are born with SMA die prematurely due to respiratory complications.

“We were thrilled with the success of our first conference webcast, and we’re very excited to be able to make this video available,” said Fight SMA President Martha Slay.  “Families facing SMA need all of the information they can get about research, and the scientists who attended our conference are some of the best in the world.”

The video features the “Basic Science Update”, one of two panel discussions during the Families and Friends Conference.  Fight SMA Science Director Dr. Chris Lorson from the University of Missouri in Columbia led the discussion.  He presented the most current information available about the search for an SMA cure.

To view and download the video, please visit the 2009 Spinal Muscular Atrophy Conference page on the Fight SMA website.  The page can be accessed via a link on the front page of the Fight SMA site, or via this link: http://www.fightsma.org/index.php?annual_meeting_2009 .

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/blog.

NEW YORK, New York – A special day-long workshop for mothers fighting diseases and health challenges that face their children will be held on Friday, May 9, 2008, from 8 a.m. to 5 p.m., at the New York Palace Hotel in New York City.

The “Fighter Mom^TM Friday” workshop is presented by Fighter Mom, a program of the nonprofit group, Fight SMA and sponsored by the Toy Industry Foundation.

Guest speaker for the day will be award-winning journalist and writer Catherine Crier, who will speak on leadership, women’s empowerment, and hope for life’s tough battles.

The workshop will provide parents who attend with techniques and tools to be advocates on behalf of their children and to work effectively to defeat the diseases and health conditions that have targeted their children.

“The information to be shared is based on more than 17 years of the experiences of mothers fighting against one particular childhood disease – spinal muscular atrophy (SMA),” said Martha Slay, event organizer and president of Richmond, VA-based Fight SMA. “Through the Fighter Mom program we can help any mom fighting any childhood disease be as effective a champion as possible. We also hope to help this special woman take good care of herself!”

“The Toy Industry Foundation is thrilled to sponsor an event like this with FightSMA to provide new insights about the value of play to parents of children with special needs,” said Jean Butler, executive director of the Toy Industry Foundation. “We have gathered experts from relevant fields to serve as resources to parents who want to do what’s best for their child and may not know exactly what that is.”

A special aspect of the day will be presentations and a discussion of the Toy Industry Foundation’s publication, “Let’s Play: A Guide to Toys for Children with Special Needs.” Toy Industry Foundation panelists will explore play and its benefits to children confined by physical and developmental barriers.

The workshop will cover such topics as developing an organizational and fundraising strategy, special events, motivating volunteers, working with scientists and doctors, lobbying Congress, and generating publicity.

Panelists for the day will include:

-Steve Eichenauer, Partner, Public Strategies Washington

-Louise Raymond, Senior Director of Global Corporate Responsibility, McGraw-Hill Companies

-Alex Mackenzie, M.D., Ph.D, Director of the Children’s Hospital of Eastern Ontario Research Institute

-Janice Schacter, chair of the Hearing Access Program at the Alexander Graham Bell Association for the Deaf and Hard of Hearing

-Diane Goetz, Director of Patient and Professional Advocacy, PTC Therapeutics

-Nancy Jeffrey, Senior Editor, People magazine

-Ellen Metrick, Toy Specialist, National Lekotek Center

-Dr. Steven Kanor, Founder and President, Enabling Devices

-Amy Jaffe Barzach, Founder, Boundless Programs; Author, Accidental Courage, Boundless Dreams

The Fighter Mom^TM manual and web site (www.fightermom.org) provide “real-world” information to help mothers (and others) not only care for their child, but to “go on the offensive” and work to eradicate the disease itself. The Fighter Mom program has been recognized by CNN and USA Today, among other media.

Fighter Mom is a program of Fight SMA, a nonprofit corporation based in Richmond, VA with chapters in 19 cities in the U.S. and Canada. Its mission is to strategically accelerate research for a treatment or cure for spinal muscular atrophy (SMA), the leading genetic cause of infant death.

The Toy Industry Foundation is a nonprofit organization whose mission is to bring joy, happiness and comfort to children in need through toys and play. While other organizations provide food, shelter and support services to children and their families, TIF sees to it that these children have toys and opportunities to play in an attempt to restore both fun and a sense of normalcy to their lives. The Foundation currently fulfills its mission through core programs including public education, The Toy Bank and PLAY HELPS. For more information, visit www.toyindustryfoundation.org.

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