Lupa –

You actually pose a very good question with regards to the data. Even though no formalized data has been released, Scholar Rock has mentioned on quite a few occasions about how muscle targeted therapies will help those of us with SMA.

All of us know that Spinraza and Evrysdi both work at the cellular level, and while there was a lot… Read more

Teri – I’ve never really heard of children suffering from seizures with regards to their Spinraza injections, but if your son didn’t have seizures before starting Spinraza, there may be a correlation.

I agree with Alyssa when she said that risdiplam will be going to a different part of the body. Since risdiplam is going to be an oral… Read more

Lupa,

I’m glad that your procedure went well. It can be somewhat unnerving to be too close to people that have tested positive. When my caregiver dropped me off at the hospital, I had to have my mask on, and I had to go through 2 different checkpoints before I was admitted into the hospital itself.

When I got to the registration desk, they… Read more

Hey Alyssa,

Everything went well. I went into the hospital about 10:45 AM, and after the procedure was completed and I had one hour to lay on my back, I was released from the hospital around 3:30 PM CST.

Hope you’re doing well, and have a blessed day.

On Friday, July 3, 2020, I had to go to UT Southwestern Medical Center in Dallas Texas to be tested for the COVID-19 virus. This test was mandatory before my next Spinraza injection scheduled for Monday, July 6, 2020.

6 hours after my test, I received notification that my results were in my UTSW Patient Portal. Even though I was… Read more

I personally don’t think that this would be considered as receiving a combination therapy. You are correct when you say that this was one of the prerequisites, and the example that I gave you regarding the gentleman that I know that had been on Spinraza and then moved to risdiplam in January 2019 is an example of this.

I don’t believe that any… Read more

Mike – I’ve never heard anything regarding a combination trial, and I personally don’t think that this would be an option during any clinical trial setting. I say this because during all clinical trials, the pharmaceutical companies are testing the efficacy and safety of a certain treatment that is trying to get approval from the FDA. I… Read more

Crystal –

My name is Michael Morale, and I’m the Senior Director of Multi-Channel Content for SMA News Today, along with their parent company, BioNews Services.

I read your comments about the pain that you’re going through with regards to your buttocks and your tailbone. I wanted let you know that there are so many of us that are going… Read more

Hi Mike – The NCT number that you gave, (NCT03032172), An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of RO7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy, is the Jewelfish trial that started back in January 2017.

If you go to http://www.clinicaltrials.gov and enter the NCT… Read more

Lupa –

It’s probably been within the last 6 to 8 months that I started noticing some strength differences in my hands, and while I do believe that Spinraza has slowed the progression of my disease, it hasn’t stopped the progression totally. I’ve talked to other people who have said the same thing regarding their condition, and given that… Read more

Derek –

I greatly appreciate you commenting on my video. My name is Michael Morale, and I’m the Senior Director of Multi Channel Content for SMA News Today, along with their parent company, BioNews Services

We just recorded a podcast where we did a roundtable discussion, and the subject of a combination therapy was brought up during… Read more

Hey everyone! Hope you’re all doing well. Check out my latest video here.

In this episode of the Morale Monologue, I discuss the importance of talking to your doctors about risdiplam. This oral therapy is extremely promising for those of us with spinal muscular atrophy (SMA). Currently, we expect the FDA to… Read more

Hey everyone! Hope you’re all staying safe and healthy out there. 

Check out my latest YouTube video here. In this episode, I discuss key differences between two potential SMA treatments. The first is Roche and Genentech’s experimental therapy risdiplam, and the second is Scholar Rock’s… Read more

 

Hey everyone! Hope you’re all staying safe and healthy out there.

In this episode of the Morale Monologue, I discuss a recent decision by the FDA regarding Roche/Genentech’s experimental SMA therapy risdiplam. Recently, Roche/Genentech submitted additional data to the FDA to… Read more

In this episode of the Morale Monologue, I’m discussing how I’m dealing with life while trying to avoid the Coronavirus. I share some of the things that I’m doing regarding keeping myself occupied, and I also share some helpful tips on what you can do to keep from feeling so isolated during… Read more

With a community full of problem solvers the forums have become a place to share ideas that will brighten spirits during these difficult times.

Also, forums Director Kevin Schaefer reflects on past times of self-isolation, and how the one he’s experiencing now… Read more

In this episode of the Morale Monologue, I’m discussing how those of us with SMA can stay safe during the Coronavirus (COVID19) concern. Those of us with SMA already have compromised immune systems in lower lung functions, and we need to be extremely careful over the next couple of… Read more

In this episode of the Morale Monologue, I’m discussing another biopharmaceutical company by the name of Scholar Rock, and their potential treatment known as SRK-015. I discuss how SRK-015 impacts our body, and how it differs from Spinraza, Zolgensma and risdiplam. While it may be over a year before this potential… Read more

Over the past 10 days, we’ve all seen an unimaginable amount of information regarding the Coronavirus. While some are staying calm and being rational with regards to keeping themselves safe, some have gone to the store and began stockpiling for what they may think will be a six-month lock-in.

While doctors and researchers tell us that… Read more

Risdiplam, in my opinion, is going to be a major player within the SMA community. Even though it will work on the SMN2 gene, similar to Spinraza, the opportunity that it provides those who are unable to take Spinraza due to medical conditions, could give these individuals the ability to start on a viable treatments for SMA.

Some of the… Read more

In this episode of the Morale Monologue, I’m discussing two important questions regarding risdiplam. Those of us that are receiving our Spinraza maintenance doses get these injections every four months. Quite a few people have said that they experience a lethargic feeling as they get closer to their next… Read more

In this episode of the Morale Monologue, I’m giving you my opinions to questions that were asked regarding Roche/Genentech’s new potential treatment known as risdiplam. These questions dealt specifically with Roche/Genentech and our insurance companies, and I believe that these are questions that will need to be… Read more

In this episode of the Morale Monologue, I’m giving you some more great information regarding Roche/Genentech’s new potential treatment known as risdiplam.

Currently, risdiplam is under Priority Review Status by the FDA, and some of my subscribers and followers to my personal… Read more

In this episode of the Morale Monologue, I’m discussing a new treatment for SMA that will possibly be available during the first half of 2020. Roche/Genentech was granted Priority Review Status, for their potential treatment known as risdiplam.

In my video, I go over a PowerPoint presentation explaining what… Read more

Hey everyone! Hope you all are doing well. For those of you in the U.S., hope you all had a great Thanksgiving.

In this episode of the Morale Monologue, I discuss how my Spinraza injections are becoming another part of my routine. For those of us who are on this treatment, many… Read more

While growing up, my parents always taught me that the animals that we had as pets were more than just pets, they were part of the family. They taught us the value that these animals bring, and by doing so, my brother and I never looked at them as animals, we thought of them as family members. While I’ve lost numerous pets in my life, I’ve… Read more

Hey everyone! Hope you’re all having a great week so far.

In this episode of the Morale Monologue, I discuss how important it is to find a physical therapist to work with you. Finding a physical therapist that’s willing to work with an SMA patient can be challenging. I share… Read more

Hey everyone! Hope you’re all having a great week so far.

Check out the latest episode of the Morale Monologue here. Recently, Biogen Pharmaceutical’s decided to conduct a study to possibly increase the dosing amounts of Spinraza. This study will look at the effects of increasing the dosing amount for those of… Read more

In this episode of the Morale Monologue, I’m sharing an email that I received from one of my subscribers to my personal YouTube channel. My subscriber asked how I continue to remain optimistic while living with SMA. I give her my reasons regarding my optimism and enthusiasm, and I’m sharing this with you in the… Read more

Happy October everyone!

In this episode of the Morale Monologue, I’m sharing a video that I did for my physical therapist, Emily. Currently, Emily and I are working on a series of articles and videos for the SMA News Today HCP site. This healthcare professional website is designed for physical therapists,… Read more

Hey everyone! Hope you all are having a great week so far.

In this episode of the Morale Monologue, I’m sharing my opinions that I gave to one of my subscribers to my personal YouTube channel. This person was having issues with a doctor, and I wanted to address these issues.

I did a video a while back… Read more

Monday, Sept. 2, Through Sunday, Sept. 08 – With everything that I’m doing at BioNews Services, along with my physical therapy schedule, and everything else that I have going on in my life, this was an extremely busy week. I’m currently working with a doctor who specializes in pulmonary rehabilitation, so we can get his podcasts started… Read more

Pawel – Cure SMA might be a good place to start, but you will want to make sure that you let them know that you live in Poland. They may be able to direct you on who you should speak to.

I hope that everything works out for you, and please keep us updated.

In this episode of the Morale Monologue, I’m sharing some information that was recently announced during the Cure SMA webinar, that was held on Wednesday, August 28, 2019. In this webinar, Cure SMA had representatives from AveXis Pharmaceuticals and the FDA. They discussed the most recent information regarding… Read more

Pawel – My name is Michael Morale, and I’m the Director of Multichannel Content for BioNews Services, the parent company of SMA News Today. I wanted to take a few minutes and try to possibly answer some of your questions and concerns. First, before I began, the ultimate decision to switch from Spinraza to Zolgensma, needs to be made between you… Read more

Monday, August 26, Through Sunday, Sept. 1 – Have you ever had one of those weeks where everything seemed to be in the wrong place just because you had to rearrange one meeting or event? My caregivers came back from vacation on Thursday morning, so I had to move my workout with Emily from Tuesday to Thursday afternoon. When Tuesday came, I… Read more

What a crazy and hectic week! My caregivers that live with me left on Tuesday for their family vacation, and I have a temporary caregiver living with me until they return. Last year, I had a bad experience with the temporary caregivers that I used. When my caregivers returned from their vacation, I was admitted to the hospital two days… Read more

In this episode of the Morale Monologue, I share a video that I produced for my physical therapist, Emily. Over the past year, Emily has been working to stretch the muscles in both of my hips.

For those of us who sit in wheelchairs for the majority of the day, these muscles in our hips tend to get tight and… Read more

Monday, August 12, Through Sunday, August 18: 

This week has been a crazy one, to say the least. A week ago, I was experiencing quite a bit of weakness in my right arm, and it persisted for some time. Luckily, my arm started to recover on Monday. My strength level then slowly improved throughout the week.

I’m not sure why I… Read more

Weekly Journal 8/5/19 through 8/11/19 – While I was hoping for an easy week, it ended up being full of activity and not much excitement. I did take a day of vacation on Friday. Thursday was my birthday, and I wanted to relax a bit. Trying to cram five days worth of work in the four days presented challenges. Still, I refused to let… Read more

In this episode of the Morale Monologue, I discuss the proper techniques to transfer a person with SMA out of their wheelchair. I’ve been doing physical therapy for the past two years. Every other week, my physical therapist and her assistant transfer me from my wheelchair to the therapy table. Physical therapists… Read more

Monday, July 29, Through Sunday, August 4 – I got so much done this week, it felt as if I had 30 hours in each day, and had eight days in the week. I guess that’s one of life’s mysteries that none of us will ever be able to fully understand. Why can we get so much done one week and then feel that we get nothing done another? It’s been… Read more

Monday, July 22, Through Sunday, July 28 – Overall, this was a great week. I completed quite a few work projects, and everything went smoothly. I ate well, got some good rest, and my body feels great. Other than a little bit of discomfort in my right hip, which I’ll talk about in just a moment, it was a pretty uneventful and… Read more

Monday, July 15, Through Sunday, July 21 – This past week was smooth and non-eventful. The only time that I left the house was when I went for my workout. Besides this, I spent the majority of my days working on podcasts, flash briefings, and trying to restructure BioNews Services’ multimedia and social media platforms. Work is keeping me… Read more

 

In this episode of the Morale Monologue, I share my tips and tricks on how to avoid getting a headache after one of your Spinraza injections. Headaches are a normal side effect with any intrathecal or spinal cord injection, and the tips that I’m sharing with you, will possibly help you to avoid… Read more

Monday, July 8, Through Sunday, July 14:

This week started out with a bang, and it got better as the week went on. On Monday, July 8, I went back to UT Southwestern Medical Center for my fifth maintenance dose of Spinraza. It’s hard to believe that I’ve now had nine injections, and it shocks me how fast the time has flown by. This… Read more

Kristin – I wanted to address your comment and maybe answer the question that you had. AveXis is currently going through clinical trials of the IT or intrathecal delivery of Zolgensma. At the present time, Zolgensma is only available to infants and toddlers 2 years of age or younger, irregardless of the type of SMA that they have. (1, 2, 3 or… Read more