Emma received a big, mysterious box today via UPS with a CureSMA label.

Inside were all kinds of neat things for Emma to play with as well as some interesting reading materials for Mark and Erin.

The items were all donated by parents and families of kids who are fighting the SMA battle. They’re items that their children found interesting, were simple to use and helped cope with the disease.

Look at all the good stuff!

A sheepskin pad, bath time toys, picture books, books with touchy-feely pages, a satin Comfort Silkie, a hand-made blanket, bubble toys, lots of rings, spoons, and grasping toys. A teddy bear, spinners, feathers, mylar balloons and a night light projector. There were even some lightweight cat toys for Emma to fight over with their cats, Gordy and Joy.

CureSMA also is involved with support groups all over the country for parents and families whose lives have been impacted by SMA.

Hopefully Emma begins her Spinraza treatments on Tuesday, 10/31… Halloween.

The following is from the Biogen pamphlet, “Unlock Her Inner Star”.

---

SPINRAZA may help improve motor milestone development

Infants taking SPINRAZA showed significant motor milestone improvements early on (day 183) in comparison with untreated infants.

ENDEAR was a well-controlled, 13-month, phase 3 clinical study with 2:1 randomization, meaning two-thirds of the individuals received SPINRAZA and one-third was untreated. The study was ended early as a result of clinically meaningful benefits seen in the planned interim analysis at day 183. All individuals were then moved into open-label extensions for further clinical study.

The interim analysis assessed the proportion of “responders,” or individuals with improvement in motor milestones, according to Section 2 of Hammersmith Infant Neurological Examination (HINE).

HINE motor milestone responders at day 183

           SPINRAZA (n=52)                       UNTREATED (n=30)

40%      Infants with milestone                            0%      Infants with milestone
improvements                                                      improvements

Ability to kick: 27%                                               Ability to kick: 0%
Head control: 33%                                                 Head control: 0%
Rolling: 35%                                                            Rolling: 0%
Sitting: 25%                                                             Sitting: 0%
Crawling: 8%                                                           Crawling: 0%
Standing: 8%                                                           Standing: 0%
Walking: 0%*                                                          Walking: 0%*

* Individuals in this study were  not of an age at which a healthy infant would typically be expected to walk.

SPINRAZA has been shown to increase the amount of SMN protein produced by SMN2 genes.

---

Remember that our little Miss Emma sports THREE SMN2 genes!

Emma and her parents visited the local North Bend Fire Department today.

They registered their house with the Department so if there’s ever an emergency at Emma’s house, the emergency crew will know all about her and will know how to get to her room.

They also were running a free baby car seat installation check for anyone who stopped by.

Emma later relaxed in her favorite chair with her favorite pumpkin.

Her grandparents baby sat while Emma’s Mom & Dad got in some Costco time.

We moved the background info on Emma’s challenge and SMA into the About link, where it belongs.

OK, OK… you can get to it here.

http://www.emmastrong2017.com/about

This is a great whiteboard presentation on SMA.

 

Here’s a good, more technical, video on Spinraza (nusinersen) and how it works.

Let me net it out for your… Spinraza works by smackin’ the SMN2 genes up side the head to smarten up and start producing more of the protein the muscle neurons need.

And remember… Emma has not one, not the usual two, but THREE of the SMN2 genes.

Don’t forget about Shannon and Michelle’s GoFundMe campaign to help with all of Emma’s upcoming expenses.

Check it out at:  http://www.gofundme.com/EmmaStrong2017.

 

Today Emma got the first half of her first flu shot.

It didn’t go over well… with either Emma or Erin.