Cure SMA-Funded Researchers Publish Paper in Journal Pediatrics

Cure SMA-funded researchers Rebecca Hurst Davis and Kathy Swoboda have published a paper, “Responses to Fasting and Glucose Loading in a Cohort of Well Children with Spinal Muscular Atrophy Type II” in the journal Pediatrics. Elizabeth A. Miller and Ren Zhe Zhang also co-authored the paper.

This study was funded by an initial $50,000 Cure SMA clinical care research grant to Dr. Swoboda.

Children with SMA sometimes develop insulin resistance or glucose intolerance, meaning they cannot properly regulate their blood sugar. The goal of this pilot project was to further understand glucose, insulin, and other responses to fasting and glucose loads in people with SMA.

The study found that children with SMA type II defined as obese using objective variables are at increased risk for impaired glucose tolerance regardless of whether or not they visually appear obese. However, further studies are needed to determine the prevalence of impaired glucose tolerance and tolerance for fasting within the broader heterogeneous SMA population and to develop appropriate guidelines for intervention.

Future Studies

In 2014, Cure SMA made a second $50,000 grant to Ms. Hurst Davis to continue research in this area. This funding will build on the results of this paper. Future studies will be used to better understand what is happening clinically in individuals with SMA, as well as to devise strategies for ways to treat patients who are insulin resistant/glucose intolerant.

Clinical Care Research

Clinical care research is part of Cure SMA’s comprehensive research funding strategy. We fund clinical care research to understand the issues that affect daily life for people with SMA, from breathing to nutrition, and to improve their quality of life today.

Earlier this year, we opened up a new clinical care research request for proposals (RFP). A clinical care RFP is an invitation for scientists to submit their best ideas for projects that address the clinical, psychological or social aspects of SMA. They explain what they want to study, how they plan to study it, and why they think it will help those affected by SMA.

Currently, those proposals are being reviewed by our Medical Advisory Council. They will carefully review all proposals to see which projects are the most intriguing, which have a well constructed study plan, and which match up with the most pressing unanswered questions about SMA. The best proposals will then receive a clinical care grant from Cure SMA.

We anticipate announcing our next round of clinical care grants in December 2015.

Pictured above: Kathy Swoboda (left) and Rebecca Hurst Davis (right).

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