SMA AWARENESS 2016 - Day 1

August is SMA Awareness Month. I will attempt to blog each day this month to tell you a little something-something about SMA, particularly how it relates to my sweet boy Colby, along with how our lives have changed since receiving his diagnosis in November 2002. Many of our family and friends know all too well the in's and out's of SMA, so much of the stuff I blog about will probably be blah blah blah to them. But maybe, just maybe, a newly diagnosed family will come across this blog and maybe, just maybe, I can provide some insight into the wild and wooley SMA world.

Today my blog is very straightforward. It concerns a possible treatment for SMA. This is exciting news, as currently there is no cure or treatment for those with Spinal Muscular Atrophy, no matter what classification (type) of SMA someone has. So basically there's this drug called nusinersen. This drug is in phase 3 of testing with SMA Type I patients. Type I is the most severe form of SMA, which happens to be the type of SMA Colby has. According to an article released by curesma.org, “the analysis found that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment.” And if I'm understanding correctly, nusinersen will soon be ready for FDA approval. This would be the first drug on the market for the treatment of SMA! And of course FDA approval takes months, if not years, to come to fruition. But hey, this is the most hope for an SMA treatment or cure I've heard of in 13.5 years! So for today, let's just be thankful a cure or treatment for this horrible condition is at least being investigated and on the horizon! Never give up hope!

What does this mean specifically for Colby? I have no idea. To the best of my knowledge there are no drug or therapy trials for older SMA Type I patients. Will he ever be considered as a candidate for a medication trial? I have no idea. Honestly, I didn't think a treatment or cure for SMA would ever come around in Colby's lifetime. But then again, if being completely honest, I never thought Colby would make it to 14 years old! So what do I know? I just know I pray daily for a cure so SMA will disappear completely and families won't have to deal with the overwhelming struggles so many other families have had, including our own. Go nusinersen, go!!!