Monday, November 28, 2016

If only I'd known.

"For the mountains may depart and the hills be removed, but my steadfast love shall not depart from you, and my covenant of peace shall not be removed,"  says the LORD, who has compassion on you. Isaiah 54:10 esv 

My friend Laura lives on the other side of our state. We try and get together as often as possible, but circumstances in our lives makes it challenging. I am a full-time caregiver for my son who suffers from Duchenne Muscular Dystrophy.  Laura not only works but also helps her dad care for her mom who is in the last stages of Alzheimer’s.  

She has supported me through some very difficult things so when she told me she was planning a 75th birthday party for her mom I wanted to be there to help her. It’s been hard for her, especially the last year, watching her mom’s abilities and memory fade away.  Not knowing if she would understand it was her birthday, it was important to Laura to make this day special for her mom.  It was her 75th Birthday and we were going to celebrate!  

When I arrived at her house Friday night, we talked about her plans for the party and what still needed to be done.

Laura was gone when I got up Saturday morning.  She left to pick up her mom as she does every Saturday.  She brings her back to her house to give her dad a break.  I was in the kitchen when I heard them come home so I walked over to say Hi. Noticing her mom did not recognize me, she still reached out her hand to squeeze mine. I gave her a little hug and then went back into the kitchen.

 A while later I heard music coming from the sun-room. I went to see what was going on and as I peeked around the corner I saw Laura sitting beside her mom at the table.  I paused in the doorway unnoticed and watched.   Her mom was eating lunch. Every few minutes Laura put food on the fork handing it to her mom while gently encouraging her to take a bite.  The music I heard was from a video of her mom’s favorite gospel music streaming on the computer.

I was surprised at my emotions as I watched them.  I thought of my own mom who lost her battle with cancer 3 years ago now watching my friend’s mom losing her battle with Alzheimer’s. Both of our moms were strong Godly women, good friends, and prayer warriors.  My mom kept a journal of people she prayed for, and Laura’s mom would often wake up in the middle of the night, get down on her knees to pray for anyone God put on her mind.  Now, she cannot speak and must be prompted to take a bite of food.

I stood there fighting back tears. The longer I watched them the more my heart filled with peace. Despite the business of party preparations Laura chose to stop and be still. To be fully engaged in the moment with her mom.  The harsh reality of the situation seemed to fade as the calm gentleness of Laura’s actions helped create an atmosphere of peace.

I eventually walked in and sat down beside them. Allowing myself to stop to be in the moment with them.

The next day I watched again as Laura went to her mom to help her up out of a chair. Gently and unhurried explaining to her what she was doing. I watched as the family gathered around her to celebrate her 75th birthday with dignity, love, and honor.

It was an emotional weekend yet I am thankful I was a part of it. I went with the assumption I was going to help and support her as she has so many times for me in the past.  But  I received more from her than what I gave. I was encouraged by watching the way she ministered to her mom.  She stopped in the flurry of life and activity to be in the moment with her mom, to minister to her mother’s heart.

Being a care-giver is hard. Caring for those we love with special needs can be exhausting. We often find ourselves rushing through the everyday task just to get them completed.  That weekend I was reminded how important it is to take the time, to slow down, to be in the moment. Focusing on the person, not just on completing the task. 

Laura has given me permission to share with you one of her journal entries. It expresses how important it is to appreciate those precious moments that can easily be overlooked. While her experience is with her mom and Alzheimer’s I believe, we can all gain some valuable insight from her words. 

If Only I'd Known;  by Laura Krieger

If only I'd known that would have been the last time I'd hear you pray by my bedside I would have tried harder to stay awake to tell you thank you after you said amen.

If only I'd known that would be the last time we would laugh and dance together I would have asked for one more song and twirled until we got dizzy.

If only I'd known that dinner you fixed would be the last time I would eat something put together by your hands I would have lingered and eaten slowly.

If only I'd known that would be the last time I would hear you sing I would have stopped what I was doing and joined you in song.

If only I'd known that would be the last time I would hear your voice telling me how much God delighted in me I would have recorded it so I could listen to it over and over and over.

If only I'd known your hug would have been the last one given freely I would have clung to you and allowed myself to feel the strength in those arms that held me as a newborn, as a teenager and as a woman.

If only I'd known that would be the last time I would hear your laughter and see your eyes sparkle with mirth I would have committed to memory what I had said to make you laugh with such abandon.

You don't have to be a care-giver to get caught up in the business of life.  Is there anything you need to let go of? Are you taking time to " be in the moment" with those who mean the most to you? 

originally posted

Thursday, November 10, 2016

A brief moment. A lasting Impression.

Psalm 103:20, eSV
Bless the Lord, you angels who belong to him, you mighty warriors who carry out his commands, who are obedient to the sound of his words.
As Veterans Day approaches I can’t help but think of a young soldier who we met for a very brief moment. His small gesture made a lasting impression on Ryan and I.  
It was near the end of the school year, and the 5th grade band which Ryan was a member was to put on a performance. This year had been a difficult year. Ryan lost a lot of physical strength and could no longer walk.The wheelchair became part of his everyday life.   He was very conscious of the wheelchair and was not ready to be on a stage in front of people. 

I understood his fears, but continued to reassure and encourage him to participate. He worked hard, in spite of his limited strength, to learn how to play the songs on the clarinet.  Although still nervous, Ryan was able to fight through extreme anxiety and fear to participated with his class on the night of the concert.  I know it was hard for him. I witnessed his battle to overcome  fear and anxiety, but he got up there and was amazing. I was so proud of him! 

After the concert while we were waiting for the gym to clear, a man in a military uniform came up from behind, tapped me on the shoulder and said, “Ma’am, can I give this to your son”? In his hand was an American flag patch that he had just removed from his uniform.  Not sure what to think I replied with a very hesitant "yes." The young soldier walked over to Ryan, bent down and shook his hand.  This young soldier proceeded to tell Ryan he did a great job, how proud he was of Ryan and that he was very brave.  While he was speaking he handed Ryan the flag. He turned to me to say good bye then headed back to where his family was waiting.  Ryan opened his hand to show me the flag.  That is when I  noticed the flag had 2 medals pinned to it. 

I knew medals were given to military personnel for specific achievements or events but did not know what these 2 medals signified.  Later that night we got on the computer to look up the significance of them.  

The parachute medal symbolized that he was assigned to an airborne unit and had participated in at least one combat parachute jump.  The second medal a Combat Action Badge, provides special recognition to Soldiers who personally engaged with the enemy or are engaged by the enemy during combat operations. The oak wreath symbolizes strength and loyalty. 

Ryan was confused. He asked, "Why did he give this to me?"  I replied, "These medals were given to him in recognition of his courage and bravery in battle. Maybe it was his way of letting  you know he sees you, and recognizes you are fighting a battle too. A different type of battle but one that also takes bravery and courage." 

This young soldier had probably gotten off of work just in time to make it to his child’s concert. Yet he felt a nudge to stop for a brief moment, to reach out and encourage Ryan.  He had no way of knowing how much fear, and anxiety Ryan battled through to get up and perform that night. Yet I have a feeling he understood Ryan’s battle far more than I could or ever will! In the same way the soldier was awarded these medals of honor for his bravery in war, he saw that Ryan deserved recognition for his bravery, not only because of what he was able to overcome that night, but in Ryan's life long battle. .       
 “The Lord gets his best soldiers out of the highlands of affliction.”  ~ Charles Spurgeon

Heavenly Father, 
I thank you that you sometimes send special angels into our lives to give us moments of  hope and encouragement. I pray a special blessing over this soldier that touched our hearts. I also pray protection and strength for all our service men and women who so willingly serve our country.
In Jesus name I pray,

originally posted

Thursday, November 3, 2016

What are you hiding behind?

Two are better than one because they have a more satisfying return for their labor; for if either of them falls, the one will lift up his companion. But woe to him who is alone when he falls and does not have another to lift him up. Ecclesiastes 4:9-10 (AMP)

I checked in for my hair appointment and was told my hairstylist was running late. I sat down in the waiting area, my mind filling with thoughts of how the day had gone so far. It had been a hard emotional day and I had no desire to be around anyone. I was in no mood to make polite small talk but knew if I canceled my appointment it would take at least a month to get back in. So I waited. I was determined once I sat in that chair, I would close my eyes and tune out the world. That was my plan anyway. 

My hairstylist finished up her client and quickly cleaned up her station. She hurried over to me apologizing for being late. I muttered, “That’s okay" and followed her to her station. I sat in the chair, and she briskly flipped the cape around my shoulders asking me how I wanted my hair cut.  I started to answer but her reflection in the mirror caught my attention. I stopped mid-sentence and said, “You look beautiful today”.  As I spoke she stopped her flurry of activity. Her eyes meet mine and started to well up with tears. She said, " Thank you. You have no idea how much that means to me.  It has been an extremely difficult week.  I just wanted to feel better so I got up extra early to make myself look presentable. I figured if I looked okay maybe I would feel better.” 

I have known her for a few years and knew she had three small boys all with some challenging issues.  I asked her a few questions about her boys, then did a lot of listening. She finished my hair, I stood up to leave and gave her a big hug! I told her I understood the feeling of being overwhelmed and reassured her, she was doing a great job with her boys! 

When I think about it, I have no idea why those words came out of my mouth. After all, I was planning on tuning out the world.  But God had other plans. He knew she needed some encouragement, someone who would listen.  In a way my plan did work out. I wasn’t going to talk, right? Well I didn't do much talking just a lot of listening. In that moment all she needed was an understanding ear.   

I wonder how we put on a “happy face”, when we are miserable on the inside.  We go to the extreme to make everything look good, like we have it all together while all the time hiding our true feelings. 

I’m not saying there is anything wrong with caring about your appearance. There are those days, even though we may not feel like it, making that extra effort does help us to feel somewhat better.  Especially when we may be in the depth of depression.   

However, it goes beyond physical appearances.  I’m thinking about when we are constantly using extra energy to hide behind the mask of “everything is great”.  The mask of perfect. The perfect family, job, house, clothes, hairstyle, marriage, etc.  If we look and act like we have it all together then maybe no one will ask questions.  We try to fool others and even ourselves into thinking if everything on the outside looks good, then everything must be great!

We are afraid of what people might think if they really knew what was going on. But keeping up this pretense is exhausting! We are using energy that we really can’t afford to keep others from truly seeing behind the mask.

I know it is hard to let your guard down and remove the mask. Not everyone can be trusted to protect your heart. Yet sometimes we need to take that chance.  Do you have a friend or two that you will allow to see what you are hiding? 

Likewise, I think we are all able to be that friend to someone else. It does take time, time for them to see you really do care and can be trusted. You can start by just being available, asking a few simple questions and really listening.

Taking the time to see what others are going through and to offer them encouragement may be just what we need as well.  By helping others, we might find the courage to drop our mask and let them in.   

Be the kind of friend that goes to the hard places that hide behind the mask.  Offering encouragement, hope and a new perspective. Life is hard. We aren’t meant to do it alone!

By yourself you’re unprotected. With a friend you can face the worst. Can you round up a third? A three-stranded rope isn’t easily snapped. Ecclesiastes 4:12 (MSG)

Heavenly Father,

Thank you for always being with us. I also know the importance of us being there for each other.  I pray that you will bring those trusted friends into our lives that we will feel safe in sharing with. Help us to offer up that same safe place for them to be open. I pray that we are able to offer encouragement and strength for each other.  

In Jesus name I pray,

Originally posted

Friday, August 12, 2016

The Power of Testimony

“In your hearts honor Christ, the Lord as holy, always being prepared to make a defense to anyone who asks you for a reason for the hope that is in you; yet do it with gentleness and respect.” (1 Peter 3:15 ESV )

I had the opportunity recently to travel to Washington DC to attend an FDA advisory committee meeting.  Over 800 parents, children and family members were in attendance that day showing support for approval of "Eteplirsen”, the first ever drug to treat Duchenne Muscular Dystrophy.

As you can imagine, emotions were high as we spent the morning and early afternoon listening to doctors and researchers presenting their charts and graphs showing the outcome of the clinical trial.  We all were hoping there would be enough evidence to convince the committee that this drug was worthy of their approval..... click on link below to continue reading...

Tuesday, June 21, 2016

Celebrating a milestone

"Give your burdens to the Lord. He will carry them. He will not permit the godly to slip and fall."
Psalms 55:22 (TLB) 

I knew it was coming. It’s been in the back of my mind for the past year. I’ve tried to avoid, ignore, distract myself but there was no way around. Once again I find myself smack dab in the middle of it. My old pal (not)…. GRIEF.

As a young boy and now a young man with Duchenne Muscular Dystrophy, Ryan’s school years were often difficult. Although a struggle, he managed to keep up academically. It was the physical, emotional and social aspects that were his hardest battles. He started displaying anxiety near the end of his elementary years and began having full blown panic attacks in junior high. By the start of high school, we withdrew him feeling homeschooling would be best for the remainder of his education. 
It was not your typical home-schooling. A tutor came to our home. Ryan kept up pretty well until this, his final year of school. His tutor moved and Ryan struggled with the change.  So we chose to do this last year on our own, in our own way. Doing so meant he would complete his education, but in a non-typical fashion.  Ryan understood that he would not graduate with his class. We talked about it and he was okay with his decision. He knew he would not “walk” with his class. There would be no pomp and circumstance. 

On the other hand, I struggled as I knew this day would come. It is hard for me to see all the postings and pictures of friend’s children at their graduations. I am happy for them. I can and will celebrate those moments with them! Yet deep down inside, it pokes at my grieving heart. 

It was Ryan’s choice. I knew I had to respect that choice. Now he is struggling too. Not because of his choice but watching his friends celebrate these events and moving on. He said,” Mom I am happy for them but my life is so different. They are talking about moving out, going to college. It is hard, I know I will never be able to be on my own.”

Trust me, we have heard all the, “You can do anything you put your mind to” speeches but the reality is he can’t and he knows that. These types of life events bring that reality to the front. You see things the way they are “supposed” to be, yet have to accept that is not our reality.

We both have to grief the loss… again.  

There are no short cuts. Each grieving moment is as painful as the last one even when you know it is coming. I cannot protect Ryan or myself from it. As much as I want to run and hide, it eventually bubbles up. We will get through it, with some tearful days. Just as we have numerous times over the past few years. No matter how many time I experience it, it does not speed up the process. We still have to walk through it.

 I am thankful God understands our sorrows and Grief as he watched his own son fulfill his destiny:
We despised him and rejected him-a man of sorrows, acquainted with bitterest grief. (Isaiah 53:3, TLB)

As we process and deal with this grief we are also choosing to see things from a different perspective. Setting aside the “normal”, we are celebrating this milestone of Ryan’s life.

The one word I could chose to describe Ryan’s life is … Endurance; The ability to do something difficult for a long time; the ability to deal with pain that continues for a long time, the quality of continuing for a long time.

Ryan endured. He endured years of watching his friends get bigger, stronger as he became weaker. He endured teachers that didn’t want to understand, IEP meetings, promises made that were never kept. He endured going from being able to walk, to needing a wheelchair. Endured having friends pull away then reject him. He endured panic of such things as there being a fire while he was on the second floor of the school, and the fear of not being able to get out. He endured disappointments, changes, challenges and pain. Most importantly, in enduring HE FINISHED WELL!

So even in the midst of our grief, we are choosing to celebrate Ryan’s unique successes. He (and our family) have learned lessons one can only learn in the school of life. Ones that often take people years to understand. 

We know that God really does care. So much so that he says in Psalms 55:22, "Give your burdens to the Lord. He will carry them. He will not permit the godly to slip and fall." (TLB) 

Perhaps Francis H. Havergal summed it up best: “To thee I bring my care, the care I cannot flee. Thou wilt not only share, but bear it all for me. O Loving Savior, now to thee, I bring the load that wearies me.”

Grief is a lonely and personal thing. Feeling  alone, yet I know I am not. Thankful that God is in it with me.  He understands, and I don’t have to hide these feelings from him or pretend everything is okay.  He can handle, " the load that wearies me".

Originally posted 6/21/2016 :


Saturday, June 18, 2016

A long awaited dream fulfilled

This past week I saw the fulfillment of a dream come true for my husband. It has been a long time in the making, 30 years to be exact.

Shortly after Jeff graduated from high school he left for Air Force basic training. He did not attend college but it was always in the back of his mind to get a degree. Early in our marriage he started taking classes at night and on weekends, while working full time. Then deployments, military training, cross country moves, the birth of 4 children, and special needs entered our world. All of these events brought additional stresses. I was overwhelmed and needed him home. At that point his dream was put on the back-burner. He chose to put the needs of our family before his desire of school. 

I know it was frustrating for him but he never made me feel bad or guilty for asking him to take a break. That desire was never taken from him, just put to the side.  As things would settled down he would take a few classes and then after 20 years he earned his BS.  He was happy with that but I knew he wanted to continue. Timing was not right.  

Then 2 years ago an opportunity came for him to return to school. We talked about what how it would work into our lives and schedules and decided to go for it. This past week he received his MBA, 30 years in the making!

Sometimes life gets in the way of our dreams and desires. Jeff persevered but never let his desires get in the way of his family’s needs.  It is hard. I know I struggle with this more than he does. I tend to want to throw a fit about it before I accept it, but he takes in stride. 

Letting go of our wants, needs and desires goes against our very nature. Yet there are times we are required to make sacrifices for those we love. Especially as a parent of a special needs child we find ourselves letting go of those things we once thought we would be or do.   

Do you have a dream that you feel will never be fulfilled? Do you trust God enough to surrender it to him? If the desire of your heart is what God desires for your life he will bring it to pass. It will not be because we have pushed ourselves to the limit or scarified our families to achieve it. It will be brought forth in his perfect timing.

If it is from God it will still be there when the timing is right. It may take 3 days or 30 years for that dream to be fulfilled. We might find as time goes by God may change our dreams and desires. He may have a whole new dream for our life. Which may go far beyond what we could have imagined. 

Trust him first. Put the needs of family above our own desires and he will honor and bless that.

“Delight yourself in the Lord and he will give you the desires of your heart”! (Psalms 37:4, NAS)

Sunday, February 7, 2016


Oswald Chambers said, “There will come one day a personal and direct touch from God when every tear and perplexity, every oppression and distress, every suffering and pain, and wrong and injustice will have a complete and ample and overwhelming explanation”.  Oh how I long for that day!

Over 2 weeks ago I flew to Washington, DC. I was part of a large group who would make a showing of support at an FDA Ad Comm meeting. To support and encourage the approval of the first ever drug for Duchenne Muscular Dystrophy, known as Eteplirsen. After our disappointing trip to California last month in hopes of Ryan being part of a different clinical trial, I was asked to submit a written testimony about our experience. The hope that my testimony, along with many others, would help the FDA to see the human side; that there are real people, boys and young men (parents included) whose lives depend on what they decide.

The wheels on the plane just touched down when my phone buzzed with incoming messages.  As we waited for the plane to disembark I scanned through the messages, stopping in disbelief when I read these words: “The FDA meeting has been cancelled due to expected inclement weather.” I re-read it another time to make sure I understood the message. We just flew 6 hours across country to attend this meeting, now what?  

Not sure what to do, I got off the plane and headed down to luggage claim. There was a shuttle driver waiting to take me and other families to the hotel. It was late and I was tired. Figured I would get to the hotel, get some sleep and then make arrangements to fly back home.

The ride from the airport to the hotel was difficult. There was a lot of traffic, the roads were icy from a trace of snow. It took over 2 hours to get to our hotel. The other families on the shuttle were in the same position as us. They had flown in from California to be part of the meeting. For 2 hours there was a lot of talk about disappointments, frustrations and making plans to return home.

We finally arrived at the hotel close to 1:00 a.m. As the families started unloading, one of the little boys was crying.  He was afraid to get off the shuttle. He knew it was icy and was afraid he was going to fall. A very real possibility for a boy with Duchennes. As the mom was struggling with luggage, I realized that the boy and his mom were traveling alone. I didn’t hesitate to ask, “Can I give him a piggy back ride into the hotel?”  He stopped crying and his mom said yes!  I turned around and told him to put his arms around my neck. I gently lifted him onto my back and down the steps into the hotel.  The moment he put his arms around my neck, the familiarity of that little action brought tears to my eyes.  I was surprised by the emotions. Not wanting to completely lose it I tried to stay focused on getting him safely inside the hotel. Still fighting to hold back the tears I walked into the lobby of the hotel and set him down on the couch. After making sure he was okay and that his mom was back with him, I walked to where my friend was waiting for me.

The moment I saw her, there was no more fighting the tears. Still confused as to why I was hit but such intense emotions I let the tears flow.  Then it hit me. The memories of Ryan being this little boy’s age and of the hundreds of times I gave him piggy back rides. I cannot begin to tell you how many times, my husband, Ryan’s older brothers and I carried him. When his legs would get tired, when the steps became too much for him to pull himself up. In fact, until we got a chair lift in our home we carried him up and down the steps in our house numerous times every single day.  

Memories are normally very positive and an important part of life. However, they can also be negative when progressive disease affects your family and friends, especially your own child. In my case, it is our youngest son Ryan who has Duchenne Muscular Dystrophy.  

I rarely look at pictures from the past. When I do I feel like I am peeking at them through my fingers, like you would when you are watching a scary show…wanting to look, but not wanting to deal with the intense emotions.  When I see pictures of Ryan walking, running, riding a bike, I know he did these things but that part of our life seems to be from a different world.  The moment he stopped walking became a forever pivotal day in our life.  Pictures are a vivid reminder of how much Ryan has lost. They show milestones to the grief that we have had to deal with.

For survival I carefully built a protective wall around the memories, to protect myself from the feelings and painful emotions they cause. But once in a while something will happen that will chip a little into that wall. That moment I lifted that boy, the familiarity of that action didn’t just chip at that wall. The wall, I worked so hard to keep in tacked, came crashing down and I was thrown back into a world that I have tried so hard to forget.

When I boarded that plane to DC I convinced myself I was just going to show support. I normally don’t like to put myself in these situations, knowing how emotional it can be, but my wall was firmly in place!  I knew about the exon skipping drug, Eteplirsen. I also knew that it did not directly affect the outcome of Ryan’s specific deletion, so I could go and just be a quiet supporter. There would be no emotions attached. That is what I told myself, until that moment.

How could I look at these younger boys and not feel? In that moment I could see how this could be a game changer for them!

Without this drug I KNOW what these families will go through. It is written in stone. I know from personal experience how much heartache, pain and grief they will have to endure over the next few years. But this drug may give them a chance! A chance to be able to have a different path in the Duchenne’s world.

Eteplirsen is not new to the scientific world. They have been working on Exon skipping as a possible treatment for 25 yrs. There has been a Patent on it for 10 years. The specific drug Eteplirsen has been in clinical trial for almost 5 years. It’s hard not to get emotional when you think about the fact they have had the technology to skip every exon for 10 years.

I think back to where my son was 10 years ago. Here is a picture of Ryan and his dad. 10 years ago, Ryan was 8. His dad giving him the very familiar piggyback ride! 

Although diagnosed at age 5, by the age of 8 he was just starting to show more intense signs of the muscle weakness. He was still able to walk, only he could not walk for very long without becoming exhausted.

There is one vivid memory I have of Ryan when he was 8 years old. He would set his own alarm to wake up extra early every morning.  He would get dressed, pour a bowl of cereal, brush his teeth and put his shoes on. He would come into my room to wake me up, give me a big hug and inform me he was ALL ready for school. Then he would ask if he could watch his favorite cartoon, “Sponge Bob”.  

Ryan is 18 yrs old now, and has to wait for me to dress him, lift him out of bed and into his wheelchair, brush his teeth and make his breakfast. He can no longer lift his arms to give me a hug. I know how hard it is for me, I can only imagine how hard it is for Ryan. He has had to grieve these losses of independence a little at a time. As he became more dependent, my once happy easy going child started battling fear and anxiety. Leaving the house to go to school became a challenge.

I think  how different it might have been when at 8 yrs old he went to MDA camp for the first time. He cried to come home after the first night.  Even after he was home he was unable to sleep for a week before he finally opened up to me.  He told me he saw older boys in wheelchairs, and some using machines to breathe. Ryan knew these boys had the same disease as he did and it scared him. We had talked a little about his disease but when he SAW those boys he came face to face with the reality of his future. No 8 year old boys should have to deal with such difficult things, such as wheelchairs and ventilators and even death at such an early age.  If only there was a drug available to him, like Eteplirsen. That conversation could have gone a whole lot different. There could have been the promise of things being different for him. It could have been a game changer in his life!

Last month we were given opportunity to go to California to be part of a clinical trial. As parents we were beyond excited and ready right then to say YES! But we knew it was Ryan’s decision to make. Ryan was understandable cautious but he said he would think about it.  He asked us a lot of questions and even did research online about it.  He finally said “Let’s go for it”.  “What do I have to lose”? 

I wish the outcome was different. Being told after Ryan’s initial screening that he didn’t meet minimal requirements for the trial was for us another loss we had to deal with.  I couldn’t help thinking if only this opportunity was presented to us 5 years ago how much different things could have been.

Eteplirsen has been in clinical trial for 5 years. The data shows the drug works, that it slows the progression of Duchenne, in some cases stops and reverses it.  The drug allows muscles to produce dystrophin, the muscle missing in Duchenne boys. The drug is completely safe. No negative side-effects, no adverse events in any of the trials. 

That is why I feel so strongly that the FDA needs to give this drug a chance. To the FDA, I will use Ryan’s own word “Let’s go for it”. “What do we have to lose?”  From the way I see it, there is nothing to lose by approving this drug but for those boys with this specific deletion they have everything to gain!

We are still waiting for the Ad Comm meeting to be rescheduled. I can only imagine how hard this is for the parents of those boys directly involved with this drug.  As I have already stated, they have been working towards this for 5 years. To get this far and then a snow storm shut the door! How discouraging and frustrating for them.

I cannot change any of that. What I can do is show my support. We are all in this together whether or not your child can benefit from this specific exon skipping drug or not. We need to stand together in support. We all know it is those with the loudest voice who gets the attention. Hopefully in this case it will be positive attention!

You don’t have to travel to Washington to have your voice heard. Here is what you can do to help.

1. Get the word out, share this with everyone you know. Share on your face book post and encourage all your friends to do the same.

2. Contact your states representatives. Encourage their support on this issue.

3.  Pray that there will be NO political games in these hearings. This is not a game, these are our children’s lives.

The day I received the call of Ryan’s diagnosis will be forever imprinted in my mind.  A phone call no parent should ever have to receive. We were in shock when we were told over the phone that Ryan had Duchenne Muscular Dystrophy. We would need to see a Neurologist to have a genetic test to confirm the diagnosis. The doctor went on to say, “There was no hurry in getting this done because there is no treatment or cure for Duchenne”. Can you imagine for a parent knowing what your childs life will be like and being told there was NOTHING you could do to change it? 

Can you imagine how much different that phone call would have been if the diagnosis of Duchennes was followed by the words” I am excited to share with you a new treatment that has just been approved by the FDA”.

Yes there IS something you can do! This drug can be a game changer for these boys! 

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