Krystal

It is a struggle for my 15-month daughter to put on weight. About two weeks ago, she was in the 10th percentile in weight for her age. She just wouldn’t gain anything! She was getting longer, but not losing or gaining, and since my husband and I are trying to get her into an upcoming clinical trial in April, my daughter needed to gain weight (she…[Read more]

Thank you for this post, it is very helpful. My daughter, bless her heart, she loves kids and babies, and is very talkative. She is only 15-months right now, so honestly all she does is blabs away in Gibberish, but hey, she’s not afraid to tell us her feelings and what’s been going on throughout her day. I hope she continues this when she begins…[Read more]

This. Absolutely this post. I just had this conversation recently with my husband. I am currently 30 weeks pregnant with a son, who will not have SMA, and am worried about when this kind of frustration will occur between him and his sister. My husband and I communicate well and we openly express ourselves, and encourage my daughter to do so (and…[Read more]

I do not have SMA but I do have a fairly large tattoo on my back that’s colored. If you have any questions about my experience you are welcome to ask! I am sure someone will be able to accommodate you 🙂

Thanks for posting this, as it will make me take a step back to remind myself that my daughter may not need help with EVERYTHING, and even at her age of 14 months, she WANTS to be more independent. My husband and I helped to make a Bumbo Wheelchair for her this past weekend, and when we placed her into the chair for the first time, she seemed to…[Read more]

My daughter is only 14 months old, so there’s no way for me to know if she thinks my husband and I are being overprotective parents. However, my husband and I have been more protective of her than before she was diagnosed. For example, before we knew she had SMA, we encouraged her to be around kids her age/kids in general as much as possible so…[Read more]

I give lots of love for your grandmother…and her name is amazing 😉

Thanks for your post. I think it’s a little outrageous that the comments on the petition came from people who did not appear to understand the difficulties, time, and money it took to make Spinraza approved in the US. However, there are parents that, when thrown into a stressful situation, may take something they first hear and run with it without…[Read more]

The shutdown may delay the enrollment date for AVXS-101 trials that are supposed to open in April. Reaching out to clinical coordinators to see if I can find a definitive answer. Thanks DeAnn for the updates!

Update: The geneticist called yesterday and said my son DOES NOT HAVE SMA. What was interesting to note is that my son actually has 3 copies of SMN1, 1 copy from me and 2 from my husband. The geneticist then explains that although my husband tested negative, the fact that our son has 3 SMN1 copies, confirm that my husband actually has 2 copies of…[Read more]

Hello, Todd. I’m sure I’ll have many questions for you regarding my daughter’s growth with SMA, as she is just a bit younger than your son and she also has Type 2. Welcome!

I would love to go, but it just depends on a lot of things, such as finances and the health of my family. I was even considering going to the conference on my own, depending on the circumstances, to network and learn. Do you think I would still  benefit from going to the conference with just me and my daughter?

My daughter received her care package a day or two after Christmas, and while I was ecstatic by the happiness my daughter was showing as I pulled out the toys one by one from the box, I was crying tears of joy on the inside. My husband and I couldn’t believe the abundance of toys and what made it even better were the little notes sent by CureSMA…[Read more]

Good morning and happy new year Jenny!

Thank you so much for your input, as it provided great information to consider. Thank you also for noting that your progression was pretty much halted between 1-3. I understand everyone is different, but it is hopeful to learn more and more from other Type 2’s and their experiences. It gives me more of an…[Read more]

Thank you so much for your response. I agree that clinical trials are just “chance” and they can be delayed and/or cancelled. This is part of the anxiety I feel when determining whether to start Spinraza or wait for a possible opportunity. At this point, we ARE in the process of getting Spinraza on board, so I feel like I’m doing something rather…[Read more]

I am a first-time mother, and also pregnant with my second, so I definitely feel like I’ve been on a roller coaster of emotions, going from being positive to be a carrier, to having my husband be negative to be a carrier, to having a positive of having the disease in my daughter, and so much more. I feel like I’ve been through so much in a short…[Read more]

Thanks for your response. I do think it’s crazy how things occur in this world, and am very glad that your son was able to get into the STRONG study that same day that you were about to get dosed with Spinraza.

It is a tough choice for sure. I know Spinraza works, but the mechanism of action is different for Spinraza vs AVXS-101. AVXS-101 is gene…[Read more]

Happy Holidays, and thank you and everyone on this forum for the warm welcome. I look forward to getting to know everyone here.