[michael-morale]

When I was growing up, my parents always taught me that nothing was free. If I wanted to make good grades in school, I had to work just as hard as anybody else. This also meant that if I needed help, that it was going to be up to me to ask for it. They taught me that asking for help was not a sign of weakness, and while they never used the term advocating for myself, this is exactly what it turned out to be.

When Kevin talked about being vulnerable with friends, I never really had a problem feeling that way because everyone that I grew up with automatically knew that I needed help doing certain things. It wasn’t uncommon for my friends to just do something for me without me asking for it, because this is the way that we were brought up. Children back in the late 60s and 70s, I think, had more empathy than the kids of today’s generation.

[michael-morale]

DeAnn –  I am exactly like you. I think for the time being, I will probably stay with Spinraza because I have seen some positive results. Once it becomes available to adults with SMA Types 2 and 3, and they have the Intrathecal delivery perfected, I’m still going to sit back and wait to see how others do on this treatment before making a decision to switch. If they show promising results, and these results would be better for me, then I would consider switching.

Lupa – ZOLGENSMA is going to be a one time injection for infants with SMA Type I, that are 9 months and younger. AveXis is currently in clinical trials for the Intrathecal (IT) delivery for those of us that are older. Infants are receiving their dosing through an IV, but, if it does get perfected for those of us that are older, it will probably be via an Intrathecal injection versus an IV. The reason why they are doing an IT delivery for adults is that the drug has to get past the blood/brain barrier. They can accomplish this through an IV for infants because of their smaller size, but adults like us, will have to have an IT administered treatment directly into the central nervous system. Another thing that you need to keep in mind, is that this is going to be a one time dose for infants, but that doesn’t necessarily mean that it will be a one time dose for those of us that are adults. That’s one of the things that they’re testing for during this clinical trial. They may find out that those of us that are adults may need more dosing, but until they finish the clinical trial, this is purely speculation on my part.

I also agree with your assessment regarding the number of copies of the SMA2 gene that you have. ZOLGENSMA is a gene therapy, whereas Spinraza is not. There will definitely be some differences between the two, and I am very hopeful that once they get past through the FDA for an IT delivery, we will probably see some outstanding results in adults like us.

[michael-morale]

Kevin, I thought the advice that you gave in your article was spot on. If this mother is a member of this forum, maybe we can get other parents who have gone through this with their kids to give her their advice and suggestions.

Great job on the article.

[michael-morale]

Thanks Adrian. I appreciate your comments.

[michael-morale]

Excellent video DeAnn.

[michael-morale]

Please be sure to take this survey.

[michael-morale]

Hey everyone, remember to take the survey.

[michael-morale]

I took the survey.

[michael-morale]

Happy Holidays to you too Kyrie.

[michael-morale]

Kyrie,

My name is Michael Morale, and I’m the Forums and Multimedia Director for BioNews Services. I was asked by Kevin Schaefer, who is the moderator of the SMA News Today forum, to respond to your question. As of right now, the projected timeframe for ZOLGENSMA to possibly be approved by the FDA as a treatment for SMA is somewhere around May or June 2019.

Once approved, which I’m sure it will be, ZOLGENSMA will only be available to infants nine months of age or under, who have SMA Type I. I know that there is also a weight limit involved, but I think that any infant that is meeting the age criteria with SMA type I will be eligible to receive this treatment. If approved, ZOLGENSMA will be an IV delivery. Results from the clinical trials were extremely impressive, but if you listen to all of the experts who talk about SMA, they will tell you that if you have an active treatment that is already available, you should go to that treatment as soon as possible because timing is crucial when it comes to the treatment of SMA, especially in young infants.

I’m not sure as to the age of your daughter, but if you consider the fact that we are still another 5 to 6 months away from the potential approval of ZOLGENSMA, your daughter would have to be less than three months of age at the present time to be accepted into this program. Also, remember that the advice that I’m giving you is only advice, and you really need to speak to your doctor regarding ZOLGENSMA and Spinraza. BioNews Services, and all of their forums that they run, are meant for informational purposes only, so please consult with your doctor to get their opinion.

Spinraza, if given to infants at an early age, has shown to be extremely effective in helping infants achieve motor milestones similar to healthy children. Of course, results will vary from child to child, but if you’re asking my opinion as to whether or not I would wait on ZOLGENSMA to be approved or to go after the treatment from Biogen, me personally, I would try to start my child on Spinraza as soon as possible because timing is crucial. Remember, these are my opinions and my opinions only.

[michael-morale]

DeAnn, that was probably one of the best videos I’ve ever seen in my life. You did such an incredible job during this interview. To hear Leah talk about her life and what she has to go through with living with SMA is something that we are probably used to ourselves, but to see her smile and to see her positive attitude really puts life in perspective.

I will definitely be subscribing to her YouTube channel and I will send her a note letting her know how much I enjoyed her interview with you.

[michael-morale]

That’s an excellent question Ryan. Fatigue comes in many shapes and sizes, and unfortunately, for those of us with SMA, fatigue can be something that is much more detrimental. I usually wake up very early in the mornings because I always had to be at work early. Now that I’m retired, I still get up early in the morning because my body is so used to it. Making sure that you’re getting enough protein in your diet and that your eating a well-balanced diet, along with getting enough sleep, can definitely help fight fatigue, but, fatigue will always be a part of our life.

Those of us with SMA tend not to take too many things for granted because we know that it can be stripped away from us very easily. Every morning that I wake up and that I don’t have bugs and dirt on my face, I’m thankful for another day. While some of my days are weak, I’m finding that the majority of my days are pretty damn good. Keeping a positive attitude and keeping your head up during the difficult times will help because I find that when I feel good about myself in the situation that I’m in, it tends to help me get through another day.

[michael-morale]

That’s exciting news to hear about your recent tests and how well you did on them. Like you, I too was somewhat skeptical when starting my Spinraza treatments, but given the amount of strength that I gained in my core and even some of the strength that I gained in my arms, I’m very thankful that I decided to go through these treatments. Hearing about your test results should be exciting news to anyone, whether they’re on these treatments are not.

I’ve always said that I tend to notice the little changes more than others, because for an able-bodied person, small changes are so insignificant, that they probably don’t even notice them. I, do notice the smaller changes because for those of us with SMA, our small changes seem to be more significant.

Congrats again on your test results and keep your head up my friend, I have a feeling that the party is just beginning.

[michael-morale]

Halsey – I’ve never actually used sheepskin before. I know my father used it when he was alive and he said that it was very comfortable to sit on. I may actually try putting some sheepskin on my wheelchair cushion and sit on it during the day to see if it would help. Have you ever tried using this in your wheelchair to sit on, and if so, do you find it helpful?

[michael-morale]

Way to go Kevin.

[michael-morale]

When they brought my cough assist to me, I told them that I wanted a mouthpiece along with the mask. The mouthpiece that they brought me was very small, but it does still work. They called me the other day and told me that they were sending me another mouthpiece that was bigger and they think that I will like it much better. When I go to my pulmonologist on Tuesday to see his respiratory nurse, I’m taking my cough assist with me so that she can verify all the settings. I haven’t tried the oscillating feature on it yet, but I’m going to talk to the respiratory nurse on Tuesday about this.

[michael-morale]

Yeah, I’m finding that using this cough assist machine is definitely going to take some getting use to. I have an appointment with my pulmonologist next Tuesday and he and his respiratory therapist are going to work on it with me. When they brought it out to me, the orders that they had from the doctor was to set the inhalation to 45. I was quite surprised at how full my lungs got whenever I am took a breath, and it was almost painful. My pulmonologist’s respiratory therapist wants me to use it twice a day to fill my lungs and to open all of the bronchial tubes. They still have to get me the proper mouthpiece because I wanted to use that instead of the mask that goes around my face and nose. When I do the cough assist using it, I am very surprised at the force that it removes the air for my lungs. As a matter of fact, the first time that I used it, I didn’t think I had any buildup in my lungs to begin with. After using it, I actually got quite a bit of phlegm out of my lungs, so I know it’s going to work.

[michael-morale]

Ryan, I really like the cough assist that I got. It’s the same one that DeAnn has and it’s also the same one that a friend of mine has that lives relatively close to me. I’ve never used one before, so I was actually surprised at how much of a help this little machine will be.

I will say this though, that little machine will suck your toenails through your lungs if you’re not careful. I wasn’t expecting it to help me exhale as strong as it did. As a matter of fact, when I used it the first time, I didn’t feel anything in my lungs to begin with, but after using it for 2 cycles, I actually got quite a bit of stuff up. I’m going to use it for a couple of weeks and then I’ll write a review about it.

[michael-morale]

You know, it’s kinda funny. When I started my Spinraza treatments back in September 2017, I thought that when I got this far into my injections, it would just be another day in my life, but I still get excited and I still get the butterflies in my stomach when I go in for each and every injection. I look at it like taking the car in for an oil change every 2000 miles. Every four months, my body gets the protein that it needs so that I can improve not only my longevity of life, but the quality of life as well.

[michael-morale]

I’ve got a heater under my desk as well. I turn that thing on in the morning when I come into my office and during the wintertime, it stays on until I go to bed. My caregivers keep their side of the house at 70°, and I keep my side of the house at 73° or 74°. Trust me, it makes for an interesting transition when I go in there to eat lunch. I come out of my warm sanctuary into the refrigerated part of the house and damn near freeze to death before I can get back in my office.

[michael-morale]

I have a backpack that I wear on my wheelchair. I’ve had this backpack for years and I put my wallet and anything that I need to carry during the day in this backpack. I tried a smaller pack similar to what you were talking about, but it was never large enough for everything that I needed to carry. The best place to get these backpacks is at an Army Navy store. These are usually well constructed backpacks that will last a very long time.

[michael-morale]

I’ve had vertigo a few times in my life. Luckily, it hasn’t happened to me while sitting in my wheelchair. It happens to me whenever I lay down and either turn to my head to my left or to my right. You can go to the doctor and they can help you with vertigo, but those of us that are in wheelchairs, don’t necessarily have this luxury because they have to put you on the table and manipulate your neck and your head to relieve the symptoms of vertigo itself. My bouts of vertigo usually only last a few days. The doctor that I spoke with said it was caused by crystals in your ear that attach themselves to the hair on the inner part of your ear next to your eardrum. After a while, these crystals do dissolve and the vertigo usually goes away in a short amount of time.

[michael-morale]

I’m exactly like you Kevin, if I’m outside too much during the wintertime, my hands tend to freeze up and it even makes it difficult for me to drive my electric wheelchair. When I go to therapy in the wintertime, I sit with the heater blowing on my hands the entire way there. I feel sorry for others who are in the van because I know I’m smoking them out given the amount of heat that I’m using, but even getting out of the van and going in to the therapy center is difficult if my hands are not already super warm. After therapy, I sit with a warm towel on my hands before I leave the building to go out to my van.

Usually, in the wintertime, I’m like you with regards to spending much of the time inside. Working full time for BioNews Services is keeping me busy enough to not go outside, so I can be thankful for that.

[michael-morale]

I’m glad you had a good birthday Kevin. Gorging yourself in food and candy on your birthday is just something that you’re supposed to do anyways, so don’t feel bad about it. I’m writing this message as you are in your physical therapy, so let us know how your stretching and your exercises went since your overindulgence on your birthday. I can speak from experience that your physical therapy probably went okay, but you may now be regretting the fact that you ate so much.

I usually go to work out on Friday with Laura, but last week, she asked me to change it to this coming Friday because they are going to get dressed up for Halloween. Instead of getting my workout with Laura, I may be being stretched out by Shrek the Ogre, and maybe some other scary creature. Either way, I don’t care if it’s Barney the dinosaur, as long as I get my workout in.

Congratulations on turning a quarter of a century old. Considering that I am twenty-eight years older than you are, you are just a pup. Have a great week.