FDA Has Approved A Drug To Treat Spinal Muscular Atrophy

Posted December 23, 2016 by Marsha in Discussion Post / 4 Comments

I do not normally put a lot of my personal life on this blog – it’s all about the books.  But right now I need to make an exception to that rule.

You see my son, who is 36, has Spinal Muscular Atrophy – if you’ve ever watched the Jerry Lewis MDA Telethon in the past, SMA is one of those diseases..

On December 23, 2016 (hours ago) the FDA approved Spinraza as a treatment drug for patients of all ages who suffer with Spinal Muscular Atrophy.  What that means to us out here in the world is a drug that will be taken every day for the rest of the patient’s life… that has the ability to reverse the affects of Spinal Muscular Atrophy.  It’s a treatment.  Is it a cure?  Depends on your interpretation of that word… to me the answer is YES.

For millions of people around the world – today is a very, very good day.

I’ve never sobbed such happy tears in my life as I am right now, even as I’m writing this.

Here is a link to the breaking news announcement that explains just why we’re so excited about this… FDA Approves Spinraza for SMA

I do not have adequate words to express my emotions right now.  Unbelievable joy and thankfulness is as close as I can get.



4 responses to “FDA Has Approved A Drug To Treat Spinal Muscular Atrophy

    • Marcy

      The possibilities of this drug are mind blowing. At the very least, it can stop SMA right where it is for my son… his heart won’t be the final muscle to stop working. At the very most possible end is he could stand for the very first time in his life. The drug has a different effect on every person depending on many factors and the effects SMA has had on the body at that point. But… the possibilities are endless. There is hope for this disease not taking lives, that children will not face the type of medically needed procedures that my son has needed over the years. Life has always been good because he’s fought the good fight… but now life has the potential to be great because now the possibilities are endless.

      I could not have gotten a better Christmas present. To stop SMA from killing my child – what more could I ever ask for.

      And, yes – there is some serious celebrating going on around here this Christmas. 🙂

      Hope you and your family have an awesome Merry Christmas!

    • Marcy

      If it does nothing more than stop the progression of the disease – I’d be happy with that. It’s hard to tell what he could expect from this as each patient has a different result… but at this time he would be content to still be able to drive his power wheelchair himself, feed himself and maybe one day get off of the ventilator. He’s not looking for walking goal – although some patients have had those results in the trials. He’ll take what ever he can from this drug and learn to live with a different mindset. The possibilities are endless.