October 2007
6 posts
FightSMA moms fight for SMA Treatment Acceleration...
On October 19th, the St. Petersburg Times ran an article about Trevor Smith and Laurie Elliot of the Tampa Bay chapter of FightSMA. Both Trevor and Laurie have urged Florida’s congressional representatives to support the SMA Treatment Acceleration Act.
From the article “Parents issue urgent plea to politicos”:
Thank God for the box of tissues. Trevor Smith couldn’t control her tears.
It could...
Fighting Back Podcast #11: SMAsquerade 2007
It’s nearly time for SMAsquerade: Make Tracks!, presented by SunTrust! The fifth annual auction fundraiser held by Fight SMA is set for the evening of Saturday, October 13. This year’s auction items are VERY exciting. The live auction, presented by Dominion, features numerous once-in-a-lifetime experiences. The silent auction includes about a dozen wonderful items as well. Plus, there’s a...
Spinal Muscular Atrophy to be featured on FOX...
Spinal Muscular Atrophy will be in the spotlight in an upcoming episode of the FOX program House. The show that will air on Tuesday, October 9 will feature an adult character with Type 3 SMA.
One of the writers for House has a seven-year-old son with Type 2 SMA, and is very active in the Southern California chapter of Families of SMA. Families of SMA, Fight SMA, and the SMA Foundation, are...
SMA Community present at two upcoming meetings
FightSMA will be hosting booth #37 at the 36th Annual Child Neurology Society Meeting being held this week in Quebec City, Quebec. Doctors and researchers will receive general information about Spinal Muscular Atrophy, current and future clinical trials, and the SMA Treatment Acceleration Act. Also, visitors to the booth will have opportunities to discuss with FightSMA Scientific Advisor Dr....
Time is running out for SMAsquerade 2007 absentee...
Fight SMA’s yearly fundraiser, SMAsquerade, is coming up on October 13, and time is running out to submit your absentee bid for some of the INCREDIBLE items we have available this year. Absentee bids must be submitted via email or fax by 5:00 p.m. on October 12. All proceeds from the auction will go toward finding a cure for spinal muscular atrophy, the leading inherited killer of children...
Fight SMA Canada to Hold SMA Conference
Fight SMA’s Canadian chapter is preparing to hold a conference of spinal muscular atrophy parents and researchers from Canada. Fight SMA Canada, otherwise known as Tori’s Buddies, is hosting the event at Bloorview Kids Rehab in Toronto. Featured speakers will include:
Dr. John Bach
Brian Weaver
Dr. Alex MacKenzie
Dr. Ben Alman
Pat McKeever
Topics on the agenda include respiratory care,...
September 2007
5 posts
FighterMom program profiled in USA Today...
We’re very excited around here this week, because the FighterMom program was profiled in an article in USA Today, the one of the nation’s largest newspapers! The story was a sidebar to an article about Shea Megale, a little girl from Northern Virginia whose stories about her assistance dog will be published and sold at FAO Schwarz.
Here’s an excerpt from the sidebar about the FighterMom...
SMAsquerade 2007 interview on “At The Races”
Fight SMA’s upcoming SMAsquerade: Make Tracks! event was featured this week in an interview on the national radio show At the Races with Steve Byk. The program airs daily on Sirius Satellite Radio channel 126, and is all about the world of horse racing. Our Steve Mullen was interviewed on Tuesday, September 25th about one of the many exciting items available during the upcoming SMAsquerade live...
Fighting Back Podcast #10 - Fighting Parry Romberg...
Welcome to episode 10 of Fighting Back, the podcast that features inspirational stories of people and families fighting serious or incurable diseases. I’m thrilled to reach this milestone episode, and we’ve found another true FighterMom, whose story is truly inspirational. 16-year-old Kelly Sperry was diagnosed with Parry Romberg Syndrome, a disease that causes a wasting of one side of the face,...
Consensus Standard of Care Released for Spinal...
We’re very excited to announce that the Consensus Statement for Standard of Care in Spinal Muscular Atrophy has been published in the August 2007 supplement of the Journal of Child Neurology. The recommendations in the document were prepared by an an international body of clinical experts. The panel worked with more than 60 spinal muscular atrophy experts in the field to achieve consensus on 5...
SMA Project RFP for Pre-IND Services - Supporting...
From the SMA Project: Dear Colleague,
We have now posted the Supporting Documentation (including the Subcontract Agreement) for the SMA Project (http://www.smaproject.org) Request for Proposals (RFP) LD-090707. This RFP seeks to identify facilities to conduct preclinical testing of chemical compounds with the ultimate goal of submitting an Investigational New Drug (IND) application to the U.S....
August 2007
5 posts
New Interventional Trial Studies Presymptomatic...
FightSMA is funding a new study called “Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants with Spinal Muscular Atrophy” or “STOP SMA” being conducted by the laboratory of Dr. Kathy Swoboda at University of Utah School of Medicine. The study will assess the safety, tolerability and potential efficacy of sodium phenylbutyrate (NaPB) in...
Fighting Back Podcast #9 - An Interview With...
This episode is a bit of a departure for the Fighting Back Podcast. We usually interview people and families fighting against serious diseases. We’ve spoken to a woman trying to raise the profile of SMA through a one-woman stage show, a mother starting a foundation to raise money for myotonic muscular dystrophy, a mother who launched what became a national organization to fight for orphan...
FDA Grants Tikvah Therapeutics, Inc. Orphan Drug...
Atlanta-based biopharmaceutical company Tikvah Therapeutics announced this week that it has received orphan drug status from the U.S. Food and Drug Administration for use of sodium phenylbutyrate as a treatment for spinal muscular atrophy (SMA). The orphan drug status gives Tikvah seven years of market exclusivity for use of phenylbutyrate for SMA treatment. Findings from in vitro studies by...
SMA Treatment Acceleration Act Introduced in U.S....
We are very pleased to announce that legislation to enhance federal support for Spinal Muscular Atrophy (SMA) research, H.R. 3334, “The SMA Treatment Acceleration Act”, has been introduced in the U.S. House of Representatives by Congressman Patrick Kennedy (D-RI) and Congressman Eric Cantor (R-VA). U.S. Senator Debbie Stabenow (D-MI) and Senator Johnny Isakson (R-GA) plan to introduce companion...
Fight SMA Releases New “Collateral Benefit”...
There’s a new page in the Fight SMA Spinal Muscular Atrophy Guidebook that anyone who is interested in orphan disease research should review. The “Guidebook” is a one-stop compilation of information on SMA. Parents of newly-diagnosed children, and even those who have known about spinal muscular atrophy for some time, can use it to answer the questions they might have about SMA. The new page in...
An Interview With Retiring NORD President and...
Abbey Meyers isn’t retiring completely, but she is going to take a little time for herself after nearly 25 years of fighting for diseases that many people didn’t seem to care about. Abbey is founder and president of the National Organization for Rare Disorders, or NORD. Recently she announced she’s stepping down from leading the group. Fighting Back, a podcast with inspirational stories about...
July 2007
8 posts
Parents Claim Some Improvement in Kids During...
The Utah chapter of Fight SMA, Dylan’s Friends, alerted us an interesting article that ran recently in the Salt Lake Tribune. At least one parent whose child is participating in a clinical trial for a treatment for spinal muscular atrophy type 2 is reporting what she believes is some success. The trial, conducted by Salt Lake City neurologist Kathryn Swoboda, is looking into using the drugs...
A New Trend Toward Playgrounds for SMA Kids and...
Two wheelchair accessible playgrounds under construction doesn’t constitute a trend, but we hope it turns into one!
There have been two recent articles from west coast newspapers about towns building wheelchair-accessible play areas, citing SMA kids as examples of children who can’t utilize traditional playgrounds.
The first article, which ran in the Seattle Post-Intelligencer, tells of a play...
SMA College Student Graduates with Perfect Grades
A truly amazing ran this week in the Catonsville (MD) Times about Kavita Krishnaswamy, who recently graduated from the University of Maryland, where she majored in computer science and mathematics. What’s amazing about the story is that she had a perfect 4.0 grade point average. In other words, she didn’t receive a grade lower than “A” during her entire college career. What’s TRULY amazing about...
SMA Dad J. Robbins Interviewed by NPR’s All Things...
Reporter Jacob Ganz interviewed Washington and Baltimore indie-rocker and producer J. Robbins this week, about his son’s battle with spinal muscular atrophy. Callum Robbins, who is nearly 18 months old, has spinal muscular atrophy type 1. The music community in DC and around the country has rallied around the Robbins family, donating charity concerts and other forms of support. More on the...
Fighting Back via Multimedia (Podcast Episode 7)
There are a number of ways to fight a serious or incurable disease. Kelly Harmsen found a rather creative and very effective technique. Her son has amblyopia, or lazy eye, a disorder that requires him to wear an eye patch for several hours every day. If his condition is not treated properly, he could lose the sight in the affected eye. The problem is, for a time he refused to wear the patch....
Nevada Girl with SMA Gets Miniature Dream Home
Make-a-wish Foundation is making the wish of a Nevada girl with SMA come true. The Reno Gazette-Journal recently published a story about seven-year-old Satori Lewis and her wish to have a playhouse big enough that she could drive her electric wheelchair inside and move around. With the help of 20 businesses in her Dayton, NV hometown, that wish will be fulfilled this week.
Here’s an excerpt...
Spinal Muscular Atrophy Drug Development Announced
Families of SMA announced recently the selection of a clinical candidate for spinal muscular atrophy through its program being conducted at deCODE chemistry. The lead compounds have shown the ability to extend survival in a mouse model of SMA.
For more information on this announcement, you can read the news release from FSMA.
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June 2007
8 posts
Article on John Bell’s Fight Against Spinal...
John Bell, or “JB”, as he’s known to his fans, is the lead singer of jamband Widespread Panic. He’s also godfather to 11-year-old Hannah Elliott of Tampa Fight SMA chapter Hannah’s Buddies, and a full-fledged member of the fight against spinal muscular atrophy (SMA). JB was profiled in a recent article in the film, culture, and music magazine “Paste”. The article, which discussed his role in...
The First Novel Clinical Candidate for Spinal...
From today’s press release:
June 22, 2007, Libertyville, IL–Families of Spinal Muscular Atrophy (FSMA) is pleased to announce the selection of a Clinical Candidate for Spinal Muscular Atrophy through its program being conducted at deCODE chemistry. At the same time FSMA is now extending its contract with deCODE to continue work towards an ...
Help with Home Improvements (Podcast Episode 6)
One thing that’s universally known by parents fighting against diseases affecting their children is that improvements to the home are usually necessary. Unfortunately, those improvements are sometimes so expensive that it’s difficult if not impossible without some assistance. Jennifer Sutton found herself in that situation, and fought back.
In episode six of Fighting Back, a podcast that...
SMA Treatment Acceleration Act to be Introduced in...
We are very pleased to announce that Spinal Muscular Atrophy legislation, “The SMA Treatment Acceleration Act”, will be introduced in both the U.S. House of Representatives and the U.S. Senate in the next few weeks.We wanted to provide each of you, SMA families, researchers and friends, with a preview of this exciting step forward for all of our individual and collective efforts to raise...
Pre-Solicitation Notice: SMA Project Funding...
From the SMA Project email service (June 8, 2007):
Dear Colleague,
I am pleased to announce the following funding opportunity for researchers in the field of spinal muscular atrophy (SMA):
The SMA Project (http://www.smaproject.org) expects to release a Request for Proposals (RFP) within the next 14 days to identify and fund facilities capable of providing the necessary research and...
Fighting Back Podcast Feedback Roundup
We’ve gotten a bit of feedback since launching Fighting Back almost two months ago, and we figure it’s time to share some of it. The podcast features inspirational stories about people and families fighting serious or incurable diseases. It’s produced for Fight SMA and FighterMom.
First up is a voice mail from a fellow podcaster. Dale from Tech Talk for Families called our feedback hotline at...
Twins With SMA Celebrate Second Birthday
A big double belated “Happy Birthday” goes out to Sam and Alex Bolton. They’re the inspiration for the British organization and website Twins With SMA. They turned two years old at the end of May.
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(via Spinal Muscular Atrophy (SMA) Blog by Fight SMA)
Assistive Technology Allows SMA Teen to Use “One...
Assistive technology has advanced by leaps and bounds, allowing those with seriously debilitating illnesses to do more. A company in the Netherlands called AssistiveWare recently released a video showing how Mchael Phillips, a teen with SMA, can play video games, instant message, and even write a book … all using only his thumb. It’s amazing technology.
Learn more about Michael on the...
May 2007
16 posts
Special Mom Fights Against Multiple Diseases...
Andrea Smith keeps busy, and she does it helping seemingly as many people as possible. She’s the latest interview subject in the Fighting Back podcast, and we’re proud to tell the story of this amazing woman.
For almost her entire life, Andrea has had epilepsy. Her young son, meanwhile, has accommodative esotropia with strabismus, an eye disorder that can lead to partial blindness if not...
Student Scientist Tackles Spinal Muscular Atrophy...
Elizabeth Heimbaugh of Akron, OH is doing research that would be impressive for scientists with years of experience. Thing is, she’s just graduating high school.
The 17-year-old recently won one of the Akron Beacon Journal Newspaper’s 2007 Gold Star Students awards. Earlier this year, she was named a semifinalist in the Intel Science Talent Search, known as the junior Nobel Prize. Her research...
Spinal Muscular Atrophy Kids Video (The Kids of...
This video slide show below was created by Jim Vaile and 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. They are the reason for our fight.
A big thanks to Jim for a great video.
families, fight sma, inspirational, kids, sma, spinal muscular atrophy, videoShare This Post Via Email or Social...
J. Robbins Speaks on Spinal Muscular Atrophy at...
Musician and SMA Dad J. Robbins gave a speech last month at a reunion benefit concert of his former band, Dismemberment Plan, last month. The speech, which was uploaded to YouTube, was about his nearly 18-month-old son Callum and the fight against spinal muscular atrophy. Fight SMA provided literature for the event to educate those in attendance.
charity events, J. Robbins, sma, spinal...
Spinal Muscular Atrophy Medical and Scientific...
On May 18, 2007, a letter signed by clinicians and investigators from the medical and scientific community was presented to the U.S. Department of Health and Human Services (HRSA) Advisory Committee on Heritable Disorders and Genetic Diseases in Newborns and Children (ACHDGDNC) restating the case for spinal muscular atrophy to be included in the newborn screening efforts. Additional signatories...
Congressman Patrick Kennedy Speaks on Spinal...
At the recent 2007 Fight SMA National Spinal Muscular Atrophy, Congressman Patrick Kennedy of Rhode Island addressed the attendees about spinal muscular atrophy research and the SMA Treatment Acceleration Act. The proposed legislation was developed through a collaborative effort between Fight SMA, the SMA Foundation, and Families of SMA, would establish a national clinical trials network to...
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Fight SMA Program FighterMom Prepares for Mother’s...
A fighter mom is a mother with a purpose. She is organized to fight a disease or disorder thats affecting her child, by raising money for research, raising awareness, and making sure everyone knows theres a horrible disease that needs our attention. She is a disease advocate. The FighterMom program salutes […] (via Spinal Muscular Atrophy (SMA) Blog by Fight SMA)
Interview with Fight SMA’s Martha Slay About the...
In Episode 4 of the Fighting Back Podcast, produced by Fight SMA and FighterMom, we continue our discussion with Fight SMA President Martha Slay. In Episode 3 we discussed the Fight SMA National Spinal Muscular Atrophy Conference in Washington D.C., as well as what it takes to be a FighterMom. In the […] (via Spinal Muscular Atrophy (SMA) Blog by Fight SMA)
Spinal Muscular Atrophy Researchers to Present...
Spinal muscular atrophy (SMA) researchers Tristan Coady and Travis Baughan, from FightSMA Science Director Dr. Chris Lorson’s laboratory at the University of Missouri, will attend and present their studies at the American Society of Gene Therapy (ASGT) Meeting in Seattle, May 30th ” June 3rd.
At this year’s ASGT meeting Travis will present, Modulating SMN2 […] (via Spinal Muscular...
N.C. Parents Visit Capitol Hill on Behalf of...
The Charlotte Observer on May 6, 2007 ran an article about the Bolton Family who started FightSMA’s North Carolina chapter, Eleanor and Jack’s Buddies, in honor of their two children who have spinal muscular atrophy. John and Holly Bolton attended the FightSMA Annual Conference in Washington D.C. in April where they visited the offices […] (via Spinal Muscular Atrophy (SMA) Blog by Fight...
An Update on the Fight Against Spinal Muscular...
Sometimes it’s hard to see turning points until you’re past them. We here at Fight SMA hope that we’re at one now. Spinal muscular atrophy is the leading genetic killer of children under two, and currently there’s no cure. However, the National Institute of Neurological Disease and Stroke (NINDS) of the […] (via Spinal Muscular Atrophy (SMA) Blog by Fight SMA)
Two New Patient Advocates Join PTC Therapeutics...
PTC Therapeutics, a company with whom Fight SMA is collaborating on research and development of a new spinal muscular atrophy treatment, announced today that they’ve added two new patient advocates. Diane Goetz and Lindsay Rosen will help advance PTC’s efforts and strengthen our collaborations with patients, families, advocacy groups, and government agencies. […] (via Spinal Muscular...
Fight SMA’s Martha Slay Discusses National Spinal...
In Episode 3 of the Fighting Back Podcast, our Steve Mullen talks with Fight SMA President Martha Slay. She discusses being the “Original FighterMom”, and the recent Fight SMA Annual Spinal Muscular Atrophy Conference, The Good Fight. This episode is the first in a two part conversation with Martha. In part two she’ll […] (via Spinal Muscular Atrophy (SMA) Blog by Fight SMA)
Progress Update from PTC Therapeutics on Spinal...
Recently, Fight SMA and the biopharmaceutical firm PTC Therapeutics announced they’re collaborating in the effort to discover new treatments for spinal muscular atrophy (SMA). PTC issued a progress update on that work today.
Read PTC’s progress update in PDF format here.
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(via Spinal...
April 2007
12 posts
Spinal Muscular Atrophy Blog Observes Day of...
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.
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(via Spinal Muscular Atrophy (SMA) Blog by Fight SMA)
Race a Duck for Spinal Muscular Atrophy
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 […] (via Spinal Muscular Atrophy (SMA) Blog by Fight SMA)