Spinal muscular atrophy (SMA) patients are often cared for by a team of specialists - neurologists, pulmonologists, orthopedists, nutritionists, etc. The Wall Street Journal reported yesterday on a shortage of pediatricians trained in specialties such as these which is causing delays in treatment for patients across the country. According to the article, “shortages of doctors across a multitude of pediatric sub-specialties are forcing 90% of hospitals to delay appointments, lose patients or refer them elsewhere” and “for families, that often means waiting for months to see a specialist and incurring heavy travel costs, often to another state.”

To read the entire article, click here.

Tags: neurology, nutrition, orthopedics, pediatrics, pulmonology, spinal muscular atrophy

Dee Horton and daughter Evie met with Senator Roger Wicker (R-MS) to thank him for cosponsoring the SMA Treatment Acceleration Act (S 1158).

FightSMA would like to thank all of the U.S. Representatives and Senators that have signed on to support the SMA Treatment Acceleration Act. Your leadership and dedication to finding a cure for spinal muscular atrophy (SMA) is admirable and appreciated by the thousands of people whose everyday lives are affected by this cruel disease.

As of August 12, 2009, the following Members have signed on as cosponsors of the SMA Treatment Acceleration Act (HR 2149, S 1158):

House of Representatives

Sponsor: Rep. Patrick Kennedy (RI) Rep. Earl Blumenauer (OR) Rep. Eric Cantor (VA) Rep. Lois Capps (CA) Rep. Yvette D. Clarke (NY) Rep. Steve Driehaus (OH) Rep. John Fleming (LA) Rep. Bob Goodlatte (VA) Rep. John J. Hall (NY) Rep. Gregg Harper (MS) Rep. Stephanie Herseth Sandlin (SD) Rep. Brian Higgins (NY) Rep. James A. Himes (CT) Rep. Steve Israel (NY) Rep. James R. Langevin (RI) Rep. Carolyn B. Maloney (NY)

Rep. Eric J. J. Massa (NY) Rep. Thaddeus G. McCotter (MI) Rep. Cathy McMorris Rodgers (WA) Rep. Mike McIntyre (NC) Rep. Gregory W. Meeks (NY) Rep. Adam H. Putnam (FL) Rep. Harold Rogers (KY) Rep. Joe Sestak (PA) Rep. Bennie G. Thompson (MS) Rep. Patrick J. Tiberi (OH) Rep. Edolphus Towns (NY) Rep. Joe Wilson (SC) Rep. Robert J.Wittman (VA) Rep. Frank R. Wolf (VA) Rep. David Wu (OR) Rep. C.W. Bill Young (FL)

Senate

Sponsor: Sen. Debbie Stabenow (MI) Sen. Sherrod Brown (OH) Sen. John F. Kerry (MA) Sen. Johnny Isakson (GA) Sen. Patty Murray (WA) Sen. Jack Reed (RI) Sen. Bernard Sanders (VT) Sen. Sheldon Whitehouse (RI) Sen. Roger F. Wicker (MS)

Don’t see your Members’ name on here? Let them know that you support the SMA Treatment Acceleration Act and they should too! Here’s 5 Ways:

1. Write a letter to your Congressman and Senators expressing your support of the bill. Need help? Click here!
2. Call your Members of Congress to let them know you support the bill. If you need contact info, call or email Caroline Gibson (804-515-0080, carolinegibson@fightsma.com)
3. Schedule a meeting with your Members in their district office. Email Caroline Gibson for help (carolinegibson@fightsma.com) Traveling to DC? Make an appointment in their Capitol Hill offices!
4. On Twitter? In just 30 seconds, you can “tweet” your Members of Congress by “Tweeting for a Cure” at http://www.EndSMA.org/twitter
5. Sign the PetitionToCureSMA.com. Over 69,000 people (and counting) have already signed!

Thank you for all you are doing to fight SMA!

Tags: congress, Mississippi, sma, SMA treatment acceleration act, spinal muscular atrophy

FightSMA has produced a slideshow of photos from the 2009 FightSMA Annual Families and Friends Conference: The Good Fight held April 27-28 in Washington, D.C.  Please check out the images below.  To read more about the event, please visit the 2009 Annual Conference page!

Thank you to our attendees, corporate sponsors, Thriving with SMA panelists, and photographer Ralph Alswang.

Tags: conference 2009, fight sma, fightsma, spinal muscular atrophy, The Good Fight, Thriving with SMA

The Temple News, a student publication of Temple University, profiled sophomore Jimmy Curran in the May 5th issue. The third of four children, Curran is the only child in his family who has type 2 spinal muscular atrophy (SMA) - a genetic neuromuscular disorder that affects motor neurons in the brain stem and spinal cord and disrupts the production of protein necessary to maintain muscle strength.

Despite being confined to a wheelchair and suffering muscle weakness, Curran hasn’t let the effects of the disease dramatically change his life. He is majoring in business at Temple University and plans to own his own business in the future. Just like any other college student, he enjoys hanging out with his friends and listening to music.

To read the entire article, click here.

Tags: college, sma type 2, SMA Type II, spinal muscular atrophy

From the University of Massachusetts Medical School E.K Shriver Center:

Help Researchers Learn About Your Emergency Preparedness Needs

Natural and man-made disasters or emergencies like severe storms, forest fires, chemical spills, floods and pandemic flu can seriously impact many people at once, including families challenged by disabilities or special health care needs.  Please help researchers understand your family’s needs related to disasters and emergencies by completing an online survey.

Researchers at the University of Massachusetts Medical School E.K Shriver Center want to learn about the emergency preparedness needs of parents of children with disabilities or special health care needs ages birth to 21.

The Shriver Center has been a pioneer in research, education and service for people with disabilities and their families for over three decades.  Your responses to this survey will assist us to develop projects and programs to meet your needs.

The brief online survey will take only a few minutes of your time. You can access the survey by going to http://www.surveymonkey.com/s.aspx?sm=fQVHbDPlA_2bfwBNT9eH8xDQ_3d_3d or pasting http://www.surveymonkey.com/s.aspx?sm=fQVHbDPlA_2bfwBNT9eH8xDQ_3d_3d
into your Internet browser window. Your responses to the survey will be completely anonymous, but essential since they will make an important contribution to the research.

For more information or questions about the survey, or to be placed on a mailing list to receive the survey results, please contact Sue Wolf-Fordham, Project Director, at susan.wolf-fordham@umassmed.edu.

Thank you for your help!

Sue Wolf-Fordham
E.K. Shriver Center
University of Massachusetts Medical School
200  Trapelo Rd.
Waltham, MA 02452-6319 USA
(tel)  781-642-0275
(fax) 781-642-0162

Tags: children with special health care needs, disability, Emergency Preparedness, survey

The National Institutes of Health (NIH) announced that Dr. Elias Zerhouni will be stepping down as NIH’s Director at the end of October. In an email to members of NIH’s extramural community, Dr. Zerhouni said, “I have had the unparalleled privilege of leading one of the great institutions in history,” and he included that he was leaving “to explore new opportunities and to devote much of my attention to writing.” Dr. Zerhouni has held the position at NIH since May of 2002.

“During his tenure, Zerhouni worked to lower barriers between disciplines of science and encourage trans-NIH collaborations,” said NIH’s press release announcing his departure. It went on to say “Zerhouni also led a major reform of the translational and clinical research system in the United States.”

To read the NIH press release and learn about Dr. Zerhouni’s accomplishments, click here.

The National Institutes of Health “is the primary federal agency for conducting and supporting basic, clinical and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases.” It is composed of 27 Institutes and Centers, including The National Institute of Neurological Disorders and Stroke (NINDS) which established the SMA Project, “a model translational research program to accelerate the process of developing a safe and effective treatment for spinal muscular atrophy.”

Tags: Elias Zerhouni, National Institute of Neurological Disorders and Stroke, National Institutes of Health, SMA Project, spinal muscular atrophy

Over the past several months, the Fight SMA team and SMArt Board have been hard at work planning a brand-new fall celebration. This revamped and more casual party will take the place of SMAsquerade, and it promises to be a highlight of the 2008 campaign. We are so excited to unveil “To Fight Life Through” to all of our longtime supporters and new friends to our cause.

Our dynamic SMArt Board is thrilled to announce that the 2008 fall celebration will be held November 15, 2008 at the stylish and beautiful BANK (1005 E. Main Street in Richmond, Va.) from 6:00-9:00pm. Tickets are $50 (tax-deductible) and valet parking is available. The party will be a dazzling evening of cocktails (including signature SMArtinis!), hors doeuvres, a jazz trio, and - most importantly - a celebration of YOU! During the party, enter the Tiffany & Co. Raffle for a chance to win one of five signature Tiffany & Co. pieces! After the party, continue to celebrate the night away at Bank, or make your way down to Shockoe’s many fabulous restaurants and bars!

We’re looking forward to celebrating with you on November 15th!

Click here to purchase tickets!

Tags: sma, smasquerade, spinal muscular atrophy

A new website has been launched to, in the words of NINDS’s Dr. John Porter, “help parents understand the how-when-where-why of clinical studies and clinical trials in their children.” While not a spinal muscular atrophy (SMA) specific site, the videos and articles address many of the concerns that parents have when considering enrolling their child in a clinical trial, including SMA trials. Topics include “Importance of Research In Kids,” “Safety and Protections,” “Questions You Should Consider Asking,” and “Effects on the Family.”

The site was developed and made possible by the support of: National Heart Lung and Blood Institute (NHLBI), NIH, National Institute of Child Health and Human Development (NICHD), Best Pharmaceuticals for Children Act (BPCA), National Marfan Foundation (NMF), National Center for Research Resources (NCRR), NIH, and Gerber Foundation.

Check out the website by visiting: www.ChildrenAndClinicalStudies.nhlbi.nih.gov.

Tags: children, clinical trial, National Institute of Neurological Disorders and Stroke, National Institutes of Health, sma, spinal muscular atrophy

Virginia Beach, Virginia’s Sawyer family is asking for five minutes … five minutes to help find a cure for SMA.  The Sawyers and their five-year-old son, Sam, appeared on local television station WAVY recently to push for support of the SMA Treatment Acceleration Act.  They’re asking for everyone to take just five minutes to write their congressional representative in support of the proposed legislation.

Here’s an excerpt of the story that appeared on the TV station’s website:

He used to be able to climb.  Now he can’t stand.  His chest muscles are also weak.  His mom says getting a common cold could kill him.

“Anytime I hear a sniffle or a little cough, I’m running him to the doctor to make sure he doesn’t have pneumonia because a little bit of pneumonia could take his life,” said Terri Sawyer.

Many children with SMA don’t live to be as old as Samuel.

Researchers say they are close to finding a cure,  but need money from the government to make it happen.

Congress is now considering  The SMA acceleration act,  and Sam’s family is asking everyone to take five minutes and write their congress members to ask for support.

You can read the entire story and watch the video that appeared on WAVY’s newscast by clicking here.  To learn more about the SMA Treatment Acceleration Act, visit our legislative information page.

You can also signal your support of the SMA Treatment Acceleration Act by signing a petition that will be sent to lawmakers.  If you haven’t signed on at the PetitionToCureSMA, please take a few minutes to do so.  It is much appreciated!

Tags: SMA treatment acceleration act, spinal muscular atrophy

The American Society of Gene Therapy, or ASGT, has posted abstracts and session details from its recent 11th Annual Meeting. Fight SMA was one of five patient advocacy groups to present at the meeting. The president of the ASGT posted a letter on the organization’s website. Here’s an excerpt:

A final highlight was the outstanding evening symposium presented by a team of five Patient Advocacy Groups. ASGT would like to welcome the ALS Association, Families of SMA, Fight SMA, the Muscular Dystrophy Association, and the SMA Foundation and we hope that our meeting will be the catalyst for productive collaborations between our members and the clinicians and patients your fine groups are representing.

You can read session overviews here, and abstracts here.

Tags: disease research, health, medical, science, sma, spinal muscular atrophy