Spinraza Treatment Begins | AJ Brockman
The long-awaited day I have been hoping for pretty much as long as I can remember has arrived. It's official, I got my first dose of Spinraza. I posted a while back on an up-and-coming drug treatment for my condition (SMA) called Spinraza. This treatment has shown amazing results in pediatric patients acting as a "cure" if administered early enough. Adults are now approved for treatment and although the impact is substantially less, it could prevent me from getting weaker and/or deteriorating further which is a huge breakthrough. After almost a year of navigating the insurance, prerequisites, genetic testing and Dr. referrals, tomorrow morning it goes down. I will have to receive this treatment every few months for the rest of my life but that is a small price to pay… The spinal tap not so much.
Spinal Muscular Atrophy (SMA)
SMA is a rare neuromuscular disorder characterised by loss of lower motor neurons and progressive muscle wasting, often leading to early death. The disorder is caused by a genetic defect in the SMN1 gene, which encodes SMN, a protein widely expressed in all eukaryotic cells (that is, cells with nuclei, including human cells) and necessary for survival of motor neurons. Lower levels of the protein results in loss of function of neuronal cells in the anterior horn of the spinal cord and subsequent system-wide atrophy of skeletal muscles.
Spinal muscular atrophy manifests in various degrees of severity, which all have in common progressive muscle wasting and mobility impairment. Proximal muscles, arm and leg muscles that are closer to the torso and respiratory muscles are affected first. Other body systems may be affected as well, particularly in early-onset forms of the disorder. SMA is the most common genetic cause of infant death.
Spinal muscular atrophy is an inherited disorder and is passed on in an autosomal recessive manner. In December 2016, nusinersen (marketed as Spinraza) became the first approved drug to treat SMA while several other compounds remain in clinical trials.
Nusinersen
Marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016, it became the first approved drug used in treating this disorder. Nusinersen has orphan drug designation in the United States and the European Union.
The drug is used to treat spinal muscular atrophy associated with a mutation in the SMN1 gene. It is administered directly to the central nervous system (CNS) using intrathecal injection.
In clinical trials, the drug halted the disease progression. In around 60% of infants affected by type 1 spinal muscular atrophy, the drug also significantly improved motor function.
Good luck to you!
Thank you!
This is great news and I am happy for you. Wishing you the best.
Thank you!
Good luck man, hope the best for you as you start treatment.
Posted using Partiko iOS
Thank you sir, really appreciate it
That's great news. I'm happy for you.
Thanks brother, will keep you in the loop.
I have thought about medical weed for treatment of my cp
Hi abrockman!
What a great post!
I'm new to steemit, and you are officially my first post for commenting, and my first 'follow'.
I joined hoping to find some health/medical content - and this certainly fits the bill!
My job is within the nursing field - so I will be keeping an eye out for future posts on how treatment is going for you.
Best of luck, and best wishes for your health!
To listen to the audio version of this article click on the play image.
![](https://steemitimages.com/640x0/https://s18.postimg.org/51o0kpijd/play200x46.png)
Brought to you by @tts. If you find it useful please consider upvoting this reply.
Congratulations @abrockman! You have completed the following achievement on the Steem blockchain and have been rewarded with new badge(s) :
Click here to view your Board of Honor
If you no longer want to receive notifications, reply to this comment with the word
STOP
Do not miss the last post from @steemitboard:
Hi @abrockman!
Your post was upvoted by @steem-ua, new Steem dApp, using UserAuthority for algorithmic post curation!
Your UA account score is currently 5.568 which ranks you at #524 across all Steem accounts.
Your rank has improved 1 places in the last three days (old rank 525).
In our last Algorithmic Curation Round, consisting of 212 contributions, your post is ranked at #71.
Evaluation of your UA score:
Feel free to join our @steem-ua Discord server