Families of SMA Funded Clinical Research Study Titled “Reliability of the Modified Hammersmith Functional Motor Scale in Young Children with Spinal Muscular Atrophy” Published in Muscle and Nerve.
February 2, 2011.

The paper published by lead author Kristin Krosschell extends the reliability of the Hammersmith Functional Motor Scale (MHFMS) to children under 30 months, allowing younger children to participate in future SMA clinical trials.

Reliable tests are needed to determine if new therapies are working in SMA when conducting future clinical trials.   Several years ago Project Cure SMA, a clinical trial network fully funded by Families of SMA, published work showing the reliability of Modified Hammersmith Functional Motor Scale (MHFMS), which estimates motor function in SMA.  This current study extends the usability of that test in clinical trials to children under 30 months of age.

Natural History data (or data collected over years from SMA patients) supports the continued loss of functional motor units with increasing age.  It also shows an increased rate of secondary complications, including contractures and scoliosis.  This can complicate clinical trials and testing of new therapies.  Additionally, recent clinical trials have suggested that children younger than 6 years of age may be better responders to therapeutic interventions. 

One prediction from this is that the expected benefit of therapies in children with SMA type II may be greater as soon as possible following initial diagnosis.  Currently a limited number of tests for clinical trials (functional outcome measures) are available to measure motor function in those with SMA Type II, younger than 2 ½ years.  Most tests that are available are not disease specific. 

This study is important as it demonstrates that a disease specific scale measuring motor function, called the MHFMS, can be reliably used in children with SMA children younger than 2.5 years of age.   This work should allow both Project Cure SMA and other clinical trial networks to include a younger age group of children with SMA, who have achieved the ability to sit independently, in both clinical trials and in clinical research studies, saidKristin J Krosschell, PT, MA, PCS, PT Principle Investigator for Project Cure SMA and Assistant Professor, Feinberg School of Medicine at Northwestern University.

In addition to testing the safety and efficacy of repurposed drugs, FSMA funding of Project Cure SMA provides for the validation and development of new outcome measures that will make future clinical trials more efficient and sensitive to effects of the drugs being studied.

Please click here to see the paper abstract.

Click here for details of FSMA funding to Project Cure SMA.

Please click here to learn about more about the clinical trial network Project Cure SMA.