Seeking a Cure for SMA
Spinal Muscular Atrophy (SMA) is the term given to a number of different disorders with a common genetic cause. They manifest themselves in weakness due to loss of the motor neurons of the spinal cord and brain stem.
Infantile SMA is the most severe form, and it’s symptoms include muscle weakness, poor muscle tone, weak cry, limpness or a tendency to flop, difficulty sucking or swallowing, and other problems. (Got all this info from Wikipedia.)
But enough of the clinical stuff: It’s just devastating to parents.
And they’re expressing their feelings on two bills here: H.R. 3334 is the SMA Treatment Acceleration Act, and S. 2042 is its Senate counterpart. The bills would require the National Institutes of Health and the Department of Health and Human Services to take various steps to coordinate programs and research trials aimed at finding treatments and cures.
They’re also telling their stories on Web sites dedicated to the young victims. Gwendolyn Strong has a Web site. And there’s also one dedicated to the memory of little Owen Shuler, who passed away in August. These children are loved.
Will these bills hasten a cure for SMA? One can’t predict easily how or how much they would help. It’s also hard to know whether this disease deserves more focus than others. But the families need outlets for their anguish, and we can all feel for them and wish them well.
vincent gill
It is to late for my grandson Owen but there are many other babies and young children who will benefit by this bill.
Owen was only 5 months old when he was taken from us by this killer. Our lives will never be the same since his death.
This bill must be passed.
Debbie Cuevas
This legislation is extremely important to the SMA kids. I am mom to Dylan who has SMA and Heather who is SMA free. My son relies on us for all physical aspects of his life. SMA kids are over the top intelligent and are sadly trapped in the wrong physical body. I run a Chapter in NY of Families of SMA and every family I meet is more dedicated than the next. We need to have National recognition of this disease. It is important for the lives of the children and adults with SMA.
erika
I think the bill should be supported because there are more and more of our children being diagnosed with this fatal disease and it’s time for the families and the children to get the support they desperately deserve and need. Thank you.
Karen Shiesley
My grandson is afflicted with this horrible disease and I pray every day that a cure or treatment is found. I do believe we are close to a cure for this genetic disease and this cure will lead to a cure for many other related and similar diseases.
Dorothy Shuler
I am Owen’s mama and honored that he was mentioned in this blog along with his long-distance girlfriend Gwendolyn. I had never heard of SMA before Owen was diagnosed on May 5. We need to make noise about this devastating disease. The fact that researchers are close to a cure and are under funded is simply unacceptable, in any economic climate. Please pass this bill. It is too late for Owen, but it isn’t for Gwendolyn and all the other children living with this horrible disease.
Diane Muoio
If recognition is not given to this dibiiating disease then there will never be any kind of future for those afflicted. I fully support continued research and development to find a cure and/or treatment for this disease and to give families hope for their children’s future. We must explore all avenues of therapy and find the funding for programs which will enhance the clinical aspects of research. Don’t give up because you will be doing a disservice to those afflicted and their families who rely on your help.
Jackie Sporbert
These bills should absolutely be passed. Proper research and funding, as well as public recognition are the keys to finding a cure for this disease.
Richard Rubenstein
The passing of these bills is important to everyone. My wife and I are parents to a beautiful 3 year old boy, Max, who is afflicted with the disease. Max is completely physically disabled and relies on us for all of his movements and positioning. Max and other children with this disease are extremely medically fragile. The common cold often becomes a life threatening condition. As a parent of an SMA child, I understand all too well the burdens the condition creates not only for the children and other parents but our society as a whole.
The medical costs relating to caring for an SMA child is staggering. Hospitalization is common place. Each child requires constant supervision of their parents and an army of health care workers. This army includes the pediatrician, pulmonologist, orthopedist, neurologist, physiatrist, 24 hour nursing, physical therapists, occupational therapists, speech therapists, and special education providers. Additionally, The care for SMA children requires specialized medical equipment, including ventilators, motorized power wheel chairs, standers, specialized seating and bedding, various braces, cough assist machines, pulse oximetry devices, suctioning devices, percussive vests, nebulizers, and gel mattresses. Often times the care for SMA children requires home modifications including lifts and elevators, ramps, and other modifications to make the home accessible including widening doorways, installing lifts and modifying bathrooms.
Obviously, the cost of all of this care is more then any family could afford and a heavy tax is levied on insurers and government agencies providing subsidies.
Finding support for a cure could lead to eliminating all of these burdens. More importantly finding a cure would provide the miracle all of the parents and our children hope and prey for on a daily basis. Until then we can only dream of the day our children can give up their ventilators and power chairs, and free themselves from the internment of their beds and take their first steps.
Dorothy Gill
Our grandson Owen Shuler passed away August 16th 2008 at five months of this horrible disease. He was dearly loved by our whole family and friends and affected so many others with his infectious smile. He put up a good fight and his parents Dorothy and Jon as all of us are truly devastated and want to keep the fight going to find a cure for SMA and will work ardently for the cause. Please pass this much needed bill for research.
Michele Erwin
This bill is important to me and family. I am a mother of a 2 year old boy who has SMA. Greyson is an adorable and charming little boy. But Like most children with SMA he can not sit, stand, crawl, or walk. Greyson will soon be recieving his first power chair. Like most children with SMA a cold can lead to complications.
Greyson needs constant care and meets with Ocupational therapists, speech specialiss, Physical therapists and doctors dailey. This is more then any child should endure.
In addition to Greyson’s needs, This illness comes with a cost to the parents, conversations about long term care, new home modifications or the purchase of a new home to accomodate all the needs for thier child. The fincially and emotional tole this puts on the parents. We need to find a cure. Our children need this bill.
Victoria Strong
This bill would benefit hundreds of diseases in the long run. Initially the SMA Treatment Acceleration Act focuses on SMA because they need to find that last missing piece of the puzzle. They know the gene responsible and why this happens, and now they just need to figure out how to change it. There are many clinical trial drugs that have already benefited diseases such as Lou Gehrig’s disease…when nothing else had previously worked. Because so much is known about SMA, scientists see the potential that a cure for SMA creates for millions of people suffering from other diseases.