The Power of Research

For those of us living with SMA, these are exciting times when it comes to research and FDA-approved treatments. I’ve always been someone who has tried to consume as much information as I can get my hands on when it comes to information regarding possible treatments and current FDA trials.

I am always checking CureSMA’s website for the SMA Drug Pipeline. This is a great resource for all approved therapies as well as those in preclinical and clinical trials.

I will then Google these therapies to read about them. Almost daily you’ll be able to find new information regarding most of these drugs.

Reading about SMA trials and therapies

I want to encourage everyone to do this. I was never interested in science when I was in high school or college.

Over the last 10+ years, I’ve been really into reading about SMA drug trials and current therapies. I have learned more about neurons, genes, backup genes, and adeno-associated virus (AAV) vectors than I would have ever dreamed of. 

The AAV vectors are fascinating to me. The hope is to one day be able to use an AAV vector to deliver gene therapy.

Before the prevalence of the internet, my main goal when I would go to see the neurologist was to learn about possible treatments. I have lived my life in the hope of a cure someday. 

Exciting advancements in treatment

Being able to hear from my neurologist about possible treatments that were being developed was always exciting. Then the Muscular Dystrophy Association started to publish Quest magazine.

If you are not on their distribution list, do yourself a favor and get on it. Quest is still a great publication for those affected with a neuromuscular disease like SMA.

Quest has always had a section on drug pipelines. SMA was always in this segment of the magazine.

Knowledge is power

The power to research and learn is now in our hands. The internet brings all of this research to our computers, tablets, or smartphones. I am a firm believer that knowledge is power.

It’s the power to make decisions about our own health journey. Obviously, all treatments have side effects and risks. It’s important to understand the risks and benefits of anything we put into our bodies.

Ask questions to educate yourself

Social media can be a slippery slope. I am a believer in personal experiences. There are numerous Facebook pages for people to discuss spinal muscular atrophy. I have found them to be a valuable resource.

Don’t be afraid to reach out in these online discussions and ask questions. At the end of the day, it’s information. I gather all of this information and then it prepares me for the conversations that I have with my neurologist.

Remember this is your body. These are all of your decisions. If you are not aware of all your possibilities, how can you make the decision that will best suit you?

Educate yourself so that you can have these discussions with your neurologist.

How do you do your own research? What’s your method? Have you ever disagreed with your doctor's recommendation?

Just remember to be open to learning and adapting as you learn new information. Don’t ever give up hope!