Run 4 a Miracle to Cure SMA

Disney's Princess Half Marathon Weekend presented by Lady Foot Locker

Fairytales do come true. One mile at a time.

Disney's Princess Half Marathon Weekend brings women of all ages together to participate in a magical event designed just for them. The Disney Princesses are the inspiration for the weekend's events and will focus on the attributes every princess possesses: commitment, courage, determination, fantasy, perseverance, and strength.

There comes a time in every woman's life when she must blaze her own trail. A time when she has to run, not from an evil stepmother, but to her moment of glory. A day when the true princess inside of her shines through. The time has come for you!

The event will take place at the Walt Disney World® Resort in Florida on a new date – February 2012, and is set to celebrate the women who prove that anything is possible in life's fairytale.

So, dust off those glass running slippers and get set for an experience nothing short of happily ever after!

I WILL BE TRAINING ALL OF 2011 FOR THE 2012 RUN 4 A MIRACLE TO CURE SMA. COME JOIN ME IF YOU CAN - IF NOT HELP ME MAKE A MIRACLE HAPPEN. MY GOAL IS TO RUN IN THE PRINCESS HALF MARATHON IN HONOR OF MY NIECE PRINCESS MADIRO AKA MADISON ROSE REED. LOOK FOR MY NEW WEBSITE COMING SOON.
-Michelle Worrellia AKA Aunt Shell

Visit mydisneymarathon.com/Princess to watch video and see photos from last year's event in the multimedia gallery.

Weekend events will include:

• Disney's Princess Half Marathon -
• Disney's Royal Family 5K
• Disney's Royal Family Kids' Races
• Disney's Fit for a Princess Expo

SMA Awareness Music Video

In support of our fellow friends in the fight; Sophia's Cure Foundation, we ask you for your help.

Upcoming singer and songwriter, 16 year old Robbie Rosen from NY heard about 1 y.o. Sophia (SMA type 1) and was inspired to write a song for her and SMA.

1. Please watch this video.

2. Comment on the video on youtube

3. Give the video a thumbs up on youtube

4. Favorite this video on youtube

5. Send an email blast out to all of your contacts

6. Share on facebook and/or twitter

7. Post on your blog or website

http://www.youtube.com/watch?v=mZ51wEIWrNk

We would like to try to get this video to go "VIRAL" in an attempt to raise SMA awareness.

Thank you for watching and for your support.

SMA Coalition Update

Some of you may remember the SMA Coalition founded to "work together to raise awareness and advocate for progress towards a treatment and cure for the leading genetic killer of infants and toddlers – SMA". Well, in recent weeks the coalition has reunited and returned to the forefront of SMA related lobbying and reaching out to government officials to ensure funding for SMA related research. Read the note below to see how YOU can become a Helping Hand in the fight against SMA!

October 26, 2010

Dear Families,

Thank you for all you have done to increase awareness and attention towards Spinal Muscular Atrophy (SMA). As most of you are aware, this has been an eventful year in the U.S. Congress; national health care and research priorities have been prominent on the radar screens of lawmakers. The significant visibility that you have brought to SMA through outreach to policymakers has yielded significant research and policy momentum for our community. Since it has been several months since we last provided a joint update on such activities, we wanted to take a moment to inform you about recent and upcoming government-sponsored, SMA-related research conferences and to provide an update on the SMA Treatment Acceleration Act (H.R. 2149 / S. 1158).

This fall, several landmark federal research meetings are being conducted to address critical regulatory and therapeutic development opportunities in SMA. In September, the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) jointly hosted a research conference focused on Antisense Oligonucleotide Therapies. If you were unable to view the live stream of this meeting, a summary will soon be available from NIH. Additionally, this week, the NIH is sponsoring a Therapy Development Workshop. For information about viewing the live stream of this week’s NIH SMA Workshop, please go here (http://www.sonicfoundry.com/NIH).

With regard to the SMA Treatment Acceleration Act, despite our best efforts and the unbelievable show of support by families across the nation, the current political climate in Washington makes it highly unlikely that the bill will pass this year. After extensive conversations over the past several weeks with senior staff persons and key Members of Congress who serve on the congressional committees with jurisdiction over the bill, it has become clear that very few, if any, public health bills such as the SMA Treatment Acceleration Act will move through Congress during the lame-duck session set to begin on November 15. Furthermore, those bills that may move have been revised considerably and we believe that weakening the SMA Treatment Acceleration Act is unacceptable to our community and is too high a price to pay in order to achieve a symbolic gain.

While we are all disappointed by this realization, we want you to know the following:

1. Even if the bill, as expected, does not pass in the lame-duck session, the overall effort to push the legislation forward has had an incredibly positive impact; it has dramatically raised awareness of SMA among lawmakers and is directly responsible for the increasing attention SMA is receiving from the National Institutes of Health (NIH).

2. We will work with our champions on Capitol Hill to evaluate and introduce a new version of the Act in the next (112th) Congress, while continuing to stay in close contact with key Members and their health staff in case circumstances change before the end of this year. Looking ahead, we will review the clinical research landscape and work with legislators to craft legislation for the 112th Congress that addresses the specific needs of the research community.While it is unlikely that the SMA Treatment Acceleration Act of 2009 will pass in the coming weeks, we believe that this is a time to celebrate the remarkable efforts of the entire SMA community in driving forward the Federal Government’s interest in SMA research. The bill has had a tremendously positive impact to date. Your efforts in securing 118 cosponsors in the House and 21 cosponsors in the Senate are responsible for the opportunities that are emerging to engage the NIH even more deeply in the effort to find a treatment for this disease.We continue to be eternally grateful to you for your efforts and hope that you will remain energetic, engaged, and hopeful as we continue in our legislative and governmental efforts. Thank you for all that you do. We would not have achieved so much without your personal contributions and outreach to Congress and we have much to be proud of. THANK YOU AGAIN for your help for all those with SMA!

Sincerely,

Kenneth Hobby Martha Slay Annie Kennedy Cynthia Joyce

Families of SMA Fight SMA MDA SMA Foundation

BELIEVE in MIRACLES!

Question & Answer Session with Dr. Kaspar

Recently Sophia's Cure Foundation had the unique opportunity to speak candidly to Dr. Brain Kaspar, one of the lead researchers on the gene therapy program at Nationwide Children's Hospital. Please view the YouTube videos below (click the link) to learn more!

Part 1 of the Q & A with Dr. Kasper
http://www.youtube.com/watch?v=s3as7-qq93U

Part 2 of the Q & A with Dr. Kasper
http://www.youtube.com/watch?v=S_QpzoSLyx4

Part 3 of the Q & A with Dr. Kasper
http://www.youtube.com/watch?v=v1z14M1yAkU

Part 4 of the Q & A with Dr. Kasper
http://www.youtube.com/watch?v=xmFTgvWAF-M

Part 5 of the Q & A with Dr. Kasper
http://www.youtube.com/watch?v=Mudffsic6Hg

BELIEVE in MIRACLES

SMA Research Team at Nationwide Children's Hospital Receives Pepsi Refresh Grant

Remember voting for Sophia's Cure Foundation in the Pepsi Refresh $250k project? Well, as you probably already know, THEY WON!!! The check was recently sent to Dr. Kaspar and his team at Nationwide Children's Hospital. Click HERE to read the press release from Nationwide Children's Hospital. Thank you to everyone who voted for this project!! We are so excited about the researcher happening here in Ohio. A Cure is Coming...

BELIEVE in MIRACLES

August is SMA Awareness Month

August is SMA Awareness Month and as such, we wanted to post videos that show part of the awareness raising that occurred in Columbus, Ohio. The videos are on YouTube and may take several minutes to load but are well worth the wait. Enjoy!

• SMA Awareness month - Proclamation -Proclamation by Mayor Tim Licklider proclaiming August as SMA Awareness month at Miracles & Madness candle lighting and balloon release on 8-11-10
• Miracles & Madness Balloon Release 8-11-10 - Watch as balloons released on the word "FLY"! Notice the light up balloons too!
  Balloon Release last day of the Miracles & Madness SMA family gathering at the Miracle League Field in Dublin, Ohio on 8-11-10. Remembering So Many Angels lost and honoring those still fighting the disease spinal muscular atrophy or SMA.
• Miracles & Madness Candle Lighting 8-11-10 -Candle lighting last day of the Miracles & Madness SMA family gathering at the Miracle League Field in Dublin, Ohio on 8-11-10. Remembering So Many Angels lost and honoring those still fighting the disease spinal muscular atrophy or SMA.

New Promotional Videos Developed for SMA Organizations

We recently decided to have our summer intern, MJ Purk, create some videos that explain SMA and feature children and adults with SMA. She will be creating four (4) versions of the video set to various types of music. We hope that these videos educate communities, individuals, corporations, and medical professionals about SMA and how we are so close to the cure with the efforts of the researchers at The Ohio State University and Nationwide Children's Hospital.

To view the Pop/Contemporary Music Version of the Spinal Muscular Atrophy Promotional Video - Click HERE
To view the Country Music Version of the Spinal Muscular Atrophy Promotional Video - Click HERE

BELIEVE in MIRACLES

Madison Featured in This Week

Can you believe it? Yep, at 13 Madison gets to make another appearance in the newspaper! The article discusses the success of the first ever Miracles & Madness Family Gathering that was put together by Miracle for Madison and was pretty successful for the first round. The article was very exciting for all of us!
Click HERE to read the article, "At 13, Dublin girl still gives hope to families facing SMA" or scroll down to read the article!



At 13, Dublin girl still gives hope to families facing SMA
ShareThisWednesday, August 18, 2010 12:20 PM
By JENNIFER NOBLIT

ThisWeek Staff Writer
Madison Reed is still fighting.

The Dublin 13-year-old born with type 1 spinal muscular atrophy has beat the odds. When she was 1, doctors told her parents to go home and enjoy what little time she had left.

According to the SMA Foundation's website, children with Madison's type of the genetic disease, characterized by progressive muscle weakening and loss, usually don't live beyond age 2.

Madison was among a group of SMA families that gathered in Dublin last week.

The gathering isn't an annual thing now, but Madison's aunt, Michellae Worrellia, said it was a good opportunity to meet other families facing the same illness.

"A long time ago, the SMA community used to do family gatherings and then stopped because we were going all over the country. It was hard to find a place for everybody to gather," she said. "We decided we wanted to do a gathering because one of the national organizations did a conference and it was in California, so we decided to get one centrally located."

Worrellia said the gathering attracted about 15 families throughout the week, and Madison's mother, Annette, said she and her daughter had the opportunity to meet people they've only spoken to online.

"We just got together and met each other," Annette said. "A lot of us have talked just over the Internet. It was nice to meet people you just talk to online."

Activities for the four-day gathering included a trip to the Mall at Tuttle Crossing, the Columbus Zoo & Aquarium and a candlelight vigil at Miracle League.

In addition to candles, the Aug. 11 event also included the presentation of a $250,000 check to local researchers, and Dublin Mayor Tim Lecklider proclaimed August as SMA month, Worrellia said.

Miracle for Madison & Friends, the Reed family's fundraising group, also has done fundraising and given money for research into cures for SMA.

"We started fundraising 12 years ago, and the Dublin community has been involved a lot," Annette Reed said. "Our fund is with the Ohio State University Foundation, and all money goes to research at OSU and at Nationwide Children's Hospital."

According to Annette, several advances have occurred recently in gene-replacement research.

"It's the most promising treatment that's out there right now, and it's to the point where we're almost there," she said, adding that FDA testing is expected to come soon. "There are some other therapies out there that look promising, but they're further away for trials. This is our best chance right now."

Though Madison has stopped attending Dublin City Schools because of health complications, Annette said, she is still fighting and celebrated her 13th birthday at home with family in February.

'We haven't been able to do a big event for a few years," she said. "I think a lot of people have forgotten what we're doing. I just want them to know Madison is still fighting."

For more information on Madison, SMA or Miracle for Madison fundraising efforts, visit miracleformadison.org.

New Research at Nationwide Children's Hospital

More SMA research out of our Ohio and some of our favorite docs, Dr. Burghes and Dr. Kaspar! Check out the latest information released by Nationwide Children's Hospital about SMA and heart problems. Also check out this podcast by Dr. Kaspar about Muscular Dystrophy, Spinal Muscular Atrophy (SMA), and gene therapy research at Nationwide Children's Hospital.

• Click HERE to read about SMA & Heart Problems - Published 8/11/2010
• Click HERE to listen to Dr. Kaspar's Podcast on Gene Therapy - Released 5/2010
  

BELIEVE in MIRACLES!

Miracles & Madness 2010 - SMA Gathering

Couldn't get to California this year for the FSMA Conference? Yearning for a more intimate gathering? Join us in Columbus, Ohio for an SMA Gathering!! We were planning the first Miracles & Madness SMA Gathering 2010!! Currently in the planning stages, we’re wondering what the interest level would be for a gathering beginning August 9th and continuing through the 12th. Currently in the works are an evening at the zoo, shopping and dinner in the Columbus area, and a meet and greet with some of the most prominent SMA researchers. We are hoping to get group rates established for at least one hotel and the zoo, so we need to know the interest level no later than July 30, 2010. Please e-mail me at mj.purk@gmail.com and let me know if you would be interested in joining us!
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Current Schedule:
Monday - Check into hotel, dinner, pool time and meet & greet with other families
Tuesday - Shopping trip to Tuttle Crossing and dinner with the researchers including Dr. Brian Kaspar, Dr. Arthur Burghes, and Dr. John Kissel
Wednesday - Free time until evening at the zoo and candle lighting
Thursday - Optional day: Some people will be traveling to KY for the Walk N Roll but you're welcome to stay in Columbus and see tourist attractions

Friends in the Fight: Introduction & July's Feature

We, at Miracle for Madison & Friends, have decided to introduce a NEW feature to raise funds for the Gene Therapy Research that is occurring at The Ohio State University (OSU) and Nationwide Children's Hospital (NCH). We're going live tonight with a new section, entitled Friends in the Fight. This idea was inspired by the mother of our first Featured Friend, Danielle, mom to Karah! Danielle came to us with this idea to raise money for the Gene Therapy Research, in hopes of enabling researchers to continue focusing on pursuing a cure for SMA.
The purpose of Friends in the Fight is to feature one individual living with SMA, one individual who passed away from SMA, and one organization that is also actively supporting Gene Therapy Research in Ohio.
This month, July 2010, we are featuring the following individuals and group. Click on their name to learn more about how YOU can make a difference in a CURE for SMA!!!!

• Karah Barry - Featured Friend
• Emma Purk - Featured Angel
• Sophia's Cure - Featured Organization
  

Help Researchers by Filling Out a Simple Questionnaire

If you or someone you know has SMA, Please take a few minutes to help researchers better understand how the Autonomic Nervous System is affected by SMA.

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Hello Everyone:
My name is MJ and I am a senior in college currently interning with Miracle for Madison and Friends. Miracle for Madison and Friends is an organization dedicated to finding a cure for Spinal Muscular Atrophy by supporting the efforts of researchers at the Ohio State University and Nationwide Children’s Hospital. As part of my internship work, I am conducting a questionnaire about autonomic nervous system problems related to SMA in children and adults with all types, including those who have earned their wings. This questionnaire is sponsored by Miracle for Madison and Friends but is not associated with any university, medical professional, or additional organizations It is currently undocumented whether or not individuals with SMA have autonomic nervous system dysfunctions and this questionnaire, although not scientifically based, will enable the medical profession to see what autonomic symptoms are most prevalent in individuals diagnosed with SMA. The questionnaire is ten (10) questions in length and should take no more than thirty (30) minutes to complete if you include additional information about your own or your child(ren)’s symptoms. If you have/had more than one child with SMA, please fill out the questionnaire for each of your children that you can recall in depth medical details about. The questionnaire will be available until August 8th 2010, but the sooner you can answer the questions, the better. At the end of questionnaire, I will compile the data and share it. There is a question asking for your e-mail address; please include your e-mail address if you wish to have the results e-mailed to you. Questions regarding this questionnaire can be directed to mj.purk@gmail.com. Again, results are *NOT* scientifically based but information gathered will be shared with several organizations, community support groups, and some medical professionals who have publicly expressed an interest in this topic, once the questionnaire is completed.

You can find the questionnaire at http://www.surveymonkey.com/s/HQLW7XK

MJ Purk
E-mail: mj.purk@gmail.com
Questionnaire: http://www.surveymonkey.com/s/HQLW7XK
Website: http://www.miracleformadison.org/

Support from Other SMA Organizations for Gene Therapy

A BIG Thank you to FightSMA/GSF for donating $250,000 towards the Gene Therapy program at Nationwide Children's Hospital. Click HERE to view the press release published by FightSMA

We also want to thank Families of SMA for their donation of $100,000 to the Gene Therapy program at Nationwide Children's Hospital. Click HERE to view the press release published by FSMA

Vote for a Cure at Pepsi's Refresh Everything

Start saving lives of children affected by Spinal Muscular Atrophy | Pepsi Refresh Everything

This is a reminder to VOTE PEPSI REFRESH for SOPHIAS CURE FOUNDATION online at:

http://www.refresheverything.com/sophiascure

Please, take a moment out of your busy schedule to vote for a cure!

How will the 250k be used? EVERY dollar will be donated to research.

Our friends in the fight- Sophia's Cure are currently in 9th place. They need to be #1 or #2 to get $250k!

At least $200k will be donated to OSU/Nationwide Children's Research Institute for the promising gene replacement program.

OSU Study: Gene Therapy Reverses Effects of Lethal Childhood Muscle Disorder in Mice

OSU and Children’s Hospital teamed up for this research. Read the Dispatch article here - Miracle for Madison is mentioned at the end. Note also the Nature article.

Thank you to Dr. Burghes and Dr. Kaspar for giving us HOPE with this latest research towards a cure for SMA. See the research article update at OSU.

This would not have been possible without each of our donors to Miracle For Madison & Friends. You are every part of this miracle!

SMA Gene Therapy

According to Dr. Kaspar, Principal Investigator, The Research Institute at Nationwide Children's Hospital: "Based on positive pre-clinical studies using a gene therapy to treat a mouse mouse model of SMA, The Kaspar and Burghes Laboratories at Nationwide Children's Hospital and The Ohio State University initiated preliminary studies in non-human primates and have seen successful translation of the gene delivery approach in a larger species."

"These results have prompted a research and clinical team including Dr. Kissel, Dr. Kolb, and Dr. Mendell to be established to initiate a translational program for human clinical trials for SMA. Presentation of the data and the plans to rapidly move the program forward are forthcoming in the next several weeks." For further information and to stay abreast of SMA research, visit our research page often.

Hocking Hills Fundraiser & Promotion

4 Seasons Hideaways in Hocking Hills, Ohio will donate 15 percent of every reservation to SMA Research if you mention Miracle For Madison when you book your stay with them. Grand Opening 10 percent discount for the month of February for any reservation in 2010. Brand new homes including an ADA accessible lodge. Check out their website at www.4seasonshideaways.com. Check out more everyday ways to help fight SMA on our events page.

4 Seasons Hideaways LLC is the home of the most environmentally friendly, short term rental lodges in the beautiful Hocking Hills of Ohio. Their cabins/lodges are the perfect destination for family reunions, family, friend, honeymoons, multiple couple, church and corporate retreat get aways.

To receive special promotional and seasonal discount offers from time to time, please become a facebook fan of 4 Seasons Hideaways! You can also register on at www.4seasonshideaways.com for a chance to win a one night free stay each year.

For more details and to ensure your discount, check out this flyer.

New SMA Presentation

On Madison's 13th birthday we were blessed with another miracle. Dr. Mary Schroth who is an SMA expert from Madison, WI spoke at Nationwide Children's Hospital on the pulmonary management of SMA. Her entire presentation can be viewed here: Respiratory Care Options For Children With Neuromuscular Weakness Thank you Dr. Schroth for taking the time out of your busy schedule to do this. Thank you Dr. John Kissel, Dr. Richard Shell & FSMA for your support.

Special Children Offer Lessons of a Lifetime by Candace Preston-Coy

Over the course of my 25 years in journalism, I've met many extraordinary people of all ages, vocations and walks of life. I've also found myself in situations that had both positive and negative effects on my life and the way I live it... Continue reading this story here.

Read more stories about Madison at Angel News.

Gene Therapy and SMA

Read the related TIME Magazine article Is Gene-Therapy Medical Treatment Ready for Prime Time?

Care Options for Neuromuscular Weakness

Read about Respiratory Care Options For Children With Neuromuscular Weakness

Meet Our Newest SMA Fan

Watch the video from our newest SMA friend and buckeye fan. This is one of the reasons we must continue to fight for a cure.

The Annual Madison's Angels at the End Zone Party

Our annual Madison's Angels at the End Zone fundraiser has been hit by several sacks, fumbles and interceptions over this past year. We must sadly inform you that we will NOT be able to host this event this year as we had previously hoped to. Our last hit and final interception came when the Buckeye Hall of Fame Cafe CLOSED it's doors on Dec. 31st, 2009. We are now without a venue for Madison's Angels at the End Zone. We have not been able to secure another comparable location which would accommodate this magnitude of an event without running into a huge financial hit.

We are still in need of a MIRACLE and ask that in HONOR of Madison's 13th birthday on 2-4-10 that you consider making a donation for $13 to recognize each year of Madison's continued fight with this horrible disease SMA! You can make an online donation by clicking on the link to the right on the sidebar entitled I-Give to OSU or please mail your check made payable to our name and address on the sidebar.

THANK YOU ALL for your support! We can't do this without YOU! Please contact us if you would like to VOLUNTEER & join us in this fight! There are many other opportunities to show your support for Madison and others with SMA. Check the events page for information on the many upcoming fundraisers and fun opportunities to get involved.

Our Heartfelt Sympathy to the Nicholson Family

One of our very special volunteers, Dorsey Nicholson, passed away recently. Dorsey is the artist who created several pieces of artwork for us that brought in several thousands of dollars for SMA Research. His most famous painting is of the Quarterbacks which highlights Rex Kern and Craig Krenzel. We are blessed to have been a recipient of his great gift! THANK YOU, Dorsey, for being one of Madison's Angels! Our hearts and prayers go out to his family. Please keep them in your prayers.

Thank You, Chris and Handel's Ice Cream!

Another one of Madison's Angels, Chris DeAngelo recently closed Handel's Ice Cream. Chris brought SMA awareness to his 3 local stores and collected tip money for MFM&F. Our heartfelt thanks goes out to Chris and his employee's for their many years of support!

New SMA Care Series Booklets

Families of Spinal Muscular Atrophy announces a new booklet in its ongoing series on SMA care. This new booklet is focused on the critical aspects of respiratory care for children with Spinal Muscular Atrophy. The booklet was authored by Mary Schroth, M.D., a member of the Families of SMA Medical Advisory Council, and a leading expert on respiratory care for SMA patients. Read more at FSMA or download a free pdf copy here.

Also available from FSMA is a new booklet on Caring Choices for Parents of Infants Newly Diagnosed with SMA Type I. Read more at FSMA or download a free pdf copy here.

Petition for SMA Treatment Acceleration Act

The Strong family from California began a petition to gather support for the SMA Treatment Acceleration Act in honor of their daughter Gwendolyn who has type 1 SMA. You can help show how important this legislation is to you by signing the petition. To sign the petition click on the End SMA sign to the left.

Here in Ohio, we have been able to secure support of this bill from Senator Sherrod Brown, Congressman Pat Tiberi and Congresswoman Debra Pryce. For this bill to go further we need more cosponsors in the House and Senate.