Cure for Cole

$6,350 of $20k goal

Raised by 105 people in 29 months
Mandy Williams  SUFFOLK, VA
Hi! I’m Cole. I’m 4 years old and I have a form of Muscular Dystrophy called Duchenne. Duchenne Muscular Dystrophy is the #1 genetic killer of young children and affects one in every 3,500 boys born worldwide.  Currently there is NO cure and the disease is 100% fatal.  There are no survivors. My muscles are missing a protein called dystrophin, which supports the muscle fibers, keeping them strong.  When a normal muscle that is being exercised would get stronger, mine gets weaker.  The natural course of the progression of the disease will have me wheelchair bound by 9 – 12 years of age, experiencing heart and lung problems next by age 15 or so and generally death will follow by late teens to early 20’s.  It is an especially brutal and cruel disease.  I have to take nasty tasting steroids for life to help slow the loss of muscle strength and I also go to Physical and Occupational Therapy at CHKD each week. I just finished my 2nd year of Preschool where I received Special Education and additional Physical, Occupational, and Speech Therapy services. I wear braces to bed at night and I am slowly adjusting to the idea of having to wear braces during the day too. I get tired easily and it’s hard for me to keep up with the other kids on the playground. I face many challenges daily like needing help to get up and down steps, and running and jumping are difficult or nearly impossible in some situations. I have just started to be able to ride a tricycle although I do need an extra push here and there to help me get going. I especially enjoy riding my motorized four-wheeler. I love feeling that wind to my face!

Research has made huge progress over the last 10 years and there are now 3 drugs that are in clinical trials that are looking very promising for the Duchenne community. With the FDA considering these New Drug Applications, my family and I are hopeful that a cure may be found in time to save me. The doctors say that my generation of boys with Duchenne may be the last to die or the first to survive….. and I want to LIVE!!!

Cincinnati Children’s Hospital Medical Center has a Neuromuscular center run by Dr. Brenda Wong, who is known throughout the Duchenne community as one of the top specialists of DMD in the country. He will be seen by Neurology, Cardiology, Pulmonology, Endocrinology, Neuropsych, have Physical, Occupational, and Speech Therapy evals, Social Work, Care Management, and a Nutrition consult. He will also have multiple lab draws and radiology studies such as an EKG, Echo, DEXA Scan, and a Holter monitor for 24 hours. They will then gather all the information together and develop a treatment and care plan and adjust his medications for him, hopefully with the goal to get him into a clinical trial. As his mother, I want him to get top notch care and treatment, giving him the best chance to live. I believe Cincinnati will give him that chance. This trip is costly and we are anticipating follow up appointments every 6 months as well as his regular appointments at home in Virginia, along with the mounting medical bills, co-pays, therapy, and medical equipment costs- and that is why I am asking for help. Cole’s father passed away from suicide when he was only 8 months old. As if that wasn’t devastating enough, Cole was diagnosed with Duchenne almost exactly 2 years later. It has been a heartbreaking last few years along with financially burdensome. Any help is appreciated. I just want to give my child a full, happy life, for as long as God will let me have him!

It only takes one muscle to make a difference- your heart!
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Update 6
Posted by Mandy Williams
2 months ago
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In case you haven't heard, today the FDA approved the first treatment (note: NOT a cure) for Duchenne! This is monumental!!! Although this drug won't benefit Cole directly, this is a step in the right direction. This means that the drug he does need will start opening clinical trial sites for enrollment. Right now it looks like DC or Baltimore are our closest options. Congrats to the Duchenne community, today is a day to celebrate!
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Update 5
Posted by Mandy Williams
2 months ago
Hi all! It's been a while since the last update, so here goes. I took Cole to Cincinnati Childrens in July where we received some bad news. Cole has now been diagnosed with Osteoporosis, caused by the steroids he has to take to preserve the level of muscle function he has left. That being said, it was discovered that he has compression fractures from T6-T9 in his spine (about midway between his shoulder blades to his middle back). Since the last update, he has also been dealing with some Gastrointestinal issues requiring additional medication. The med they usually use to help with Osteoporosis can't be used on him since he's having GI issues, as it can erode his esophagus. This past week he completed his first round of IV Pamidronate to treat the Osteoporosis. They are four hour long infusions that he has to have 3 days in a row every 3 months. Side effects are flu-like symptoms. He has been having migraines and complaining of lower back pain since the infusions, but today only had a headache. His mobility is beginning to worsen as it's harder for him to go up and down steps and fatigues easily when playing with his peers. It's heartbreaking. I know that the "honeymoon" period is over and this is now the beginning of the decline of Duchenne. We are making plans to make our home wheelchair accessible. Unfortunately I know this needs to happen sooner rather than later due to his added diagnosis's. He is repeating Kindergarten and has started on medication for ADHD. This kid can't catch a break! He always has a smile on his face though, which just amazes me. He's such an incredible little boy. I'll leave you with a picture I took last Tuesday at the hospital while he was getting his infusion. Even after a blown IV, he's got a smile on his face. Hope he puts one on your face too! Thanks for following his story and continuing to support us throughout this journey. Please share his page and help raise awareness of Duchenne Muscular dystrophy!
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Update 4
Posted by Mandy Williams
16 months ago
We went to Cincinnati again a few weeks ago and Cole is maintaining, which is good news. His heart function is still good which was a relief. He remains on steroids (Deflazacort) that we get from London and the side effects have been much better on this than Prednisone.
We found out today that the local firefighters union is sending Cole and I to Disney for a week this Sunday. He is so excited! I can't wait to see his face when he meets Mickey! Thank you to everyone for your prayers and support. You all are truly a blessing to us!
Riding the fire truck
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Update 3
Posted by Mandy Williams
29 months ago
Cincinnati day 3 done! What an exhausting 3 days this has been! Cole had thoracic and lumbar x-rays in the morning. He was a little wiggly worm, but they managed to get decent pics. He has a slight curvature of the spine (boys with DMD are prone to getting scoliosis) but will continue to monitor. Had the follow up meeting with the Neuropsych MD who recommended intensive speech therapy. He's got a lot of language/ communication execution delays that they can help with. She did give me a gold star in handling his tantrums (in telling him that I recognize he's upset & tired, but has to work through it and finish his tests; commanding him to do something shuts him right down). She also gave some tips for his upcoming IEP meeting with the public school system for this school year. The day continued on, meeting with the genetics counselor, social worker (who was actually able to help me answer a question I've been asking for the last 2 years!) & nutritionist (who was very impressed with how much milk he drinks and strawberries he eats!). He was seen by Cardiology who reviewed his test results with us and there are some mild things that will need to be closely monitored, but all in all, his heart function is in good health for now. (Yay!!!) We reviewed some final recommendations from Dr. Wong, but the best news was hearing that the drug he would need is coming up for clinical trials next year!!! The FDA is expanding eligibility criteria for these trials to children younger than 7, meaning Cole will have a chance! {cue the angels singing} Dr. Wong will notify us when a trial becomes available that she feels will be beneficial to him and that he meets eligibility for. She did say that the travel expenses for the trials may not be covered by whoever is funding the trial. Another roadblock, but when there's a will, there's a way.

Cole was a trooper through all of that, so I took him for a treat to the Cincinnati Zoo. He loved it! His favorite was the polar bear. He growled at it and told him to wake up! We met up with our other Duchenne friends for dinner and goodbyes.

I am so happy I didn't sit back and listen to the Doctor who said "Take him home & love him, there's nothing we can do". There may not be a cure at this moment, but there is treatment to help prolong his life and make it the best quality it can be. We'll be back to Cincinnati in January.

Thank you everyone for your continued support. This is a long, expensive road ahead of us. Any help is appreciated! Please continue to share Cole's story and raise awareness of Duchenne Muscular Dystrophy.
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Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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$6,350 of $20k goal

Raised by 105 people in 29 months
Created July 09, 2014
KS
$50
Kitty Smith
2 months ago

Miss seeing you every week Mandy. Cole looks happy and you are an amazing mom

BP
$50
Burma Pavidus
2 months ago
$50
Anonymous
2 months ago
KH
$62
Kara Hearn
2 months ago
$50
Anonymous
2 months ago
KM
$100
Kathleen McNeely
2 months ago
$50
Anonymous
2 months ago
$25
Theresa O'Connor
2 months ago
TC
$50
Tonya Clay
2 months ago
KL
$50
Klaudia Luli
2 months ago
CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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CR
Cheryl Riley
29 months ago

God Bless you Cole & Mandy!

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