On Monday, April 30, 2007, the blogosphere will observe a day of silence to remember the victims of the Virginia Tech massacre. Fight SMA and the Spinal Muscular Atrophy (SMA) Blog are observing this day.
If you’re in the Westfield, New Jersey area, or even if you’re not, you can participate in an event that puts the “fun” in “fundraiser”. Patrick’s Buddies, a chapter of Fight SMA based in New Jersey, is holding the “Fight SMA Rubber Duck Race” on Sunday, May 6, from 11 a.m. to 2 p.m. to benefit spinal muscular atrophy research and Fight SMA. The 25 fastest ducks in the race at Mindowaskin Park in Westfield will receive an iPod shuffle (all the better to listen to the Fighting Back Podcast!) Ducks are $5 each, or a “Quack Pack” of 5 ducks for $20. If you want to improve your chances, a “Flock” of 12 Ducks is only $50, and a “Gaggle” of 25 ducks for $100. For more information, contact Ellen Devin at ej-devin@comcast.net.
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The name and focus of the Spinal Muscular Atrophy Podcast, which made its debut last week, have changed. We have decided to integrate stories from FighterMom into the mix. The new focus of the podcast is stories about people and families fighting any serious or incurable disease. The new name is the Fighting Back Podcast, which reflects our dedication to families fighting orphan and other serious diseases. Stories about SMA will still be told, but if you’re a regular listener, you’ll hear about a wide variety of people who are fighting their own fight every day.
In the latest episode of the Fighting Back Podcast (which we’re calling episode 2 since Natasha Wood’s story falls squarely into the new podcast description) our Steve Mullen interviews Edibell Stone. Mrs. Stone is fighting Myotonic Muscular Dystrophy, along with much of the rest of her family. She recently found the FighterMom program by Fight SMA, which provides support and advice for mothers who have become disease advocates for their children. In her own words, we hear about Mrs. Stone’s family and about her fight against the disease.
If you have a comment or question for the Fighting Back Podcast, or are interested in us telling your story or the story of someone you know, told in the Fighting Back Podcast, please contact us via the Fight SMA contact page, or leave us a message at 206-984-3669. Of course, you can always leave a message in the form of a comment on this blog as well.
To hear episode 2 of the Fighting Back Podcast, you have several options. You can listen using the Flash mp3 player above. Or, if you’re a fan of podcast RSS feeds, you can use the Fighting Back Podcast Feed in your favorite aggregator. Finally, you can of course download this episode, using the link above.
Technorati Tags: fightermom, incurable diseases, myotonic muscular dystrophy, orphan diseases, podcastAfter opening with a cocktail reception the night before, the FightSMA Annual Conference hosted a full day of researcher presentations on Monday at the L’Enfant Plaza Hotel in Washington, D.C. FightSMA Science Director Dr. Christian Lorson kicked off the day at 8 o’clock in the morning with a review of the basic science of spinal muscular atrophy (SMA). This was followed by eleven scientists who represented the United States, Canada, Germany, Italy, the National Institutes of Health, university researchers, and private sector biotechnology corporations. With the contributions of the additional scientists and researchers in the audience, this diverse group openly discussed published and unpublished research, recent successes and failures, and novel tactics in the fight against SMA. The energy of the room was evident as the attendees looked towards the second day of presentations on Tuesday.
Technorati Tags: annual conference 2007, biotechnology, National Institutes of Health, neuromuscular, research, scientists, sma, spinal muscular atrophy, washington dcFamilies from all over the nation are traveling to the nation’s capital with a goal of advancing the fight against the deadly disease spinal muscular atrophy (SMA). The families will be in Washington D.C. from April 22-27 to attend the twelfth Fight SMA Annual Spinal Muscular Atrophy Conference, The Good Fight, where they will learn about the disease, network with researchers and other SMA families, and work toward legislation to assist in finding a cure for spinal muscular atrophy.
Spinal muscular atrophy is an incurable neuromuscular disorder that kills more infants than any other genetic disease. SMA affects the motor neurons of the spinal cord and brain stem, rendering them unable to communicate with muscle cells properly. The National Institute of Neurological Disease and Stroke (NINDS) of The National Institutes of Health (NIH) says, however, that out of the 600 disorders it tracks, SMA is one of the closest to being cured. Fight SMA is an international nonprofit organization dedicated to accelerating that cure.
Many of the families in attendance will meet with their Senators and Representatives to urge them to support the creation of the first spinal muscular atrophy legislation in our country’s history. The proposed SMA Treatment Acceleration Act, developed through a collaborative effort between Fight SMA, the SMA Foundation, and Families of SMA, would establish a national clinical trials network to develop treatments that are safe and effective for SMA patients
“The conference attendees are able to explain better than anyone why the fight against spinal muscular atrophy is so critical,” said Fight SMA President Martha Slay. “We know these meetings with members of Congress will put a face on this horrible disease.“
Attendees will also have the opportunity to receive updates on research into the disease from scientists from all over the world, gain valuable and practical advice from clinicians for coping with a childhood disease on a daily basis.
For more information on the conference, please visit the Fight SMA 2007 Annual Conference web page.
Technorati Tags: conference 2007, cure, families, researchers, sma, spinal muscular atrophy, treatment
Welcome to Fight SMA’s first entry into the world of podcasting. We spoke recently to Natasha Wood (pictured to the left), who has spinal muscular atrophy and is currently starring in her own one-woman autobiographical show in North Hollywood, California. The show, dubbed Rolling with Laughter, covers her life from her diagnoses at birth, to a childhood selling women’s lingerie with her parents, to her decision to go on tour to raise her profile leading up to a performance in London next spring to benefit the UK SMA charity Jennifer Trust.
In the podcast, Fight SMA’s Steve Mullen introduces us to Natasha, and we hear in her own words the inspirational story of how she has never let her disease slow her down.
Technorati Tags: california, inspirational, interview, podcast, sma, SMA Type II, spinal muscular atrophyFight SMA has a new partner in its search for a treatment or cure for spinal muscular atrophy (SMA). The international nonprofit organization has established a research and development collaboration with PTC Therapeutics, Inc (PTC) to identify and develop a compound that can be used to treat SMA. SMA is the leading genetic cause of infantile death, yet there currently is no treatment available. PTC Therapeutics, Inc. is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, and has made great strides toward developing compounds for a variety of genetic disorders including cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD).“This is an exciting opportunity to push toward a treatment for SMA,” said Dr. Christian Lorson, PhD, Scientific Director of FightSMA. “The unique genetic context of SMA is well-suited for the type of therapeutic intervention in which PTC Therapeutics specializes, and with the highly skilled scientists at PTC, we are hopeful that great strides can be made towards identifying a drug for this devastating disease.”
“We’re thrilled that PTC Therapeutics is committed to the fight,” said Fight SMA President Martha Slay. “The team at PTC is dedicated and experienced. Theirs is an incredible combination of talent and determination to see these therapies delivered to children and adults suffering with SMA.”
PTC Therapeutics, Inc. is also a sponsor of the annual international Fight SMA research conference, The Good Fight. The conference will be held in Washington, DC from April 22nd to the 24th.
Technorati Tags: health, research, scientists, sma, spinal muscular atrophySickle cell anemia and spinal muscular atrophy are genetic diseases that are serious, but relatively uncommon.
Sickle cell anemia is an inherited blood disorder that affects about 70,000 people in the United States, mostly blacks. Spinal muscular atrophy is a motor-neuron disorder that affects about 1 in 6,000 newborns.
[New company President and CEO Michael] Herr, who came to Orphagenix from the University City Science Center, a Philadelphia incubator for early-stage life science and technology companies, says both diseases are well-suited to the gene-replacement process Kmiec and Parekh-Olmedo developed.
The two researchers found a way to introduce a minuscule fragment of DNA into a cell where a portion of the DNA is damaged.
The introduced DNA binds to the damaged portion of the DNA, triggering the cell to heal itself. What is introduced into the cell is eventually released out of the cell.
The article credits the Orphan Drug Act, passed in 1983, to Orphagenix’s interest in SMA. The law provides a host of incentives, from a streamlined review process to a tax credit that allows companies to recoup as much as 50 percent of their research-and-development costs.
You can read the entire article by clicking here.
Technorati Tags: delaware, research, researchers, sma, spinal muscular atrophyMothers fighting on behalf of their children can be unstoppable. Erin Weisner of Albuquerque, New Mexico is such a mother, and it’s because of her hard work that people all over her area are learning about spinal muscular atrophy (SMA) via a series of billboards.Ms. Weisner’s daughter, Desiree, has spinal muscular atrophy, a neuromuscular disease that kills more children under two than any other genetic disorder. To battle the disease, Ms. Weisner founded Desiree’s Buddies / Fight SMA Southwest, an Albuquerque-based chapter of Fight SMA. Fight SMA is an international non-profit organization dedicated to accelerating a cure for spinal muscular atrophy.
Six months ago, Ms. Weisner went to her local office of Lamar Outdoor Advertising and asked for a price quote from the company to put up billboards to inform people about the deadly disease. She told the sales manager Desiree’s story and invited him to learn about SMA. In the end, Lamar agreed to donate space on 20 billboards. Desiree’s Buddies was only charged the cost of printing the posters applied to the billboards themselves.
“We’re thankful to Lamar for everything they’re doing to help us,” said Ms. Weisner. “Billboards are a great way to get the word out, and I hope people will see them, take the time to visit the Fight SMA website, and get involved.”
The billboards feature a simple black background with white block lettering. They inform people viewing them that, “Spinal muscular atrophy kills more babies than any other genetic disease.”
To view an image of one of the billboards, go to the Desiree’s Buddies/Fight SMA Southwest chapter page.
This story is also available via:
PRLeap: Spinal Muscular Atrophy (SMA) Billboards Debut in Albuquerque, New Mexico
PR Log: Spinal Muscular Atrophy (SMA) Billboards Debut in Albuquerque, New Mexico
A Richmond, Virginia spinal muscular atrophy family and great supporter of Fight SMA is holding a fund raiser on May 19 (that’s next month!) to help their little girl. “Taylor’s Trot”, a 5K walk, run, or roll, will raise money to go toward a companion dog for Taylor, a 19-month-old girl with SMA. Taylor was diagnosed with spinal muscular atrophy type 2 in September of 2006, just a couple of weeks shy of her first birthday. As you can see from the picture to the left, she’s a beautiful little girl. The companion dog will come from an organization called 4 Paws for Ability and will cost $9,800. The dog will be Taylor’s constant companion, helping her with many different daily activities.
The 5K will begin at James River High School at 8:30 a.m. The registration fee is $20. More information, including how to participate and/or donate, can be found at www.ourthreegirls.com. Please check it out and help Taylor!
Technorati Tags: companion dog, fundraiser, sma, SMA Type II, spinal muscular atrophyiTunes Users: Visit the Fighting Back Podcast Store!
