Update on Brielle and Brooke - Clinical Trial Visit


is in a clinical trial at the University of Utah called STOP SMA under the direction of Dr. Swoboda. For this trial we visit every 3 months.

There are three specific things she has to undergo each visit:

Electromyography Test (EMG) testing: It records the electrical activity from the brain and/or spinal cord to a peripheral nerve root (found in the arms and legs) that controls muscles during contraction and at rest.

a. Specifically compound motor action potential (CMAP) and Motor Unit Number Estimation (MUNE) scores.

DEXA Bone Density Scan

Physical Therapy Test: Using the Hammersmith Functional Motor Scale For Children With Spinal Muscular Atrophy

Since our last visit in March 2010 Brooke has increased her CMAP and doubled her MUNE scores! This is really exciting because she had a BIG drop in March so Dr. Swoboda has been able to recover some of that motor neuron loss. Brooke’s nutrition has been a major factor in this. The feeding tube has really helped her gain weight and strength the past 3 months. She scored very high on the physical therapy test.


is part of the sibling/compassionate study with Dr. Swoboda and is seen once a year so this was her second visit to the clinic. She goes through all the same testing that Brooke does and also did very well on the physical therapy test. Her bone density has increased 22% which is exceptional and we attribute that to all the standing she does and walking in her gait trainer and her Standing Dani. The Standing Dani is an excellent piece of equipment that really helps Brielle get standing time in and be able to “run” and keep up with her friends outside. Her EMG showed that her CMAP and MUNE scores have dropped quite a bit but we are hoping to recover some of the loss through an increase in medications. Dr. Swoboda reminded us that her rapid growth can cause the loss in motor neurons like what happened this time and she has grown quite a bit in the past year! We have to look at the overall picture of how well she is doing and keep trying our best to keep up with all the physical therapy/nutrition and medications so she can be in the best shape she can possibly be in.

More info about testing can be found here: http://smaoutcomes.org/hammersmith_manual/manual_1.html