Cure SMA Attends the 71st Annual American Academy of Neurology (AAN) Meeting in Philadelphia

By Cure SMA | Published On May 20, 2019

The American Academy of Neurology (AAN) 2019 Annual meeting wrapped up in Philadelphia, Pennsylvania on May 10th, 2019. The AAN Annual meeting is one of the world’s largest professional meeting of neurologists on cutting-edge science, research, and clinical updates. Over 14,000 neurology professionals and meeting attendees were in attendance at the meeting highlighting more than 3,000 posters and platform presentations on the latest research in neurology. Scientific leadership from Cure SMA were in attendance at AAN to promote awareness for SMA through the new SMArt Moves campaign and to share outcomes from the Cure SMA annual surveys and a treatment algorithm for infants diagnosed with SMA through newborn screening.

SMArt Moves Booth

Cure SMA participated as an exhibitor at the 2019 American Academy of Neurology Annual Conference in order to raise awareness about SMA and spread the word on the importance of early diagnosis and treatment. There the SMArt Moves campaign was shared with attendees. SMArt Moves, is a disease awareness and educational campaign developed by Cure SMA to empower parents and healthcare professionals to promptly recognize and diagnose the early signs of SMA.

Cure SMA works with health care professionals (HCP), and specialists to ensure that patients affected by spinal muscular atrophy (SMA) have access to available treatment(s) and the recommended standard of care. One of our key educational goals is to reduce the diagnostic delay in SMA and raise awareness about the urgency of early diagnosis and treatment particularly, in the most severe of SMA types. To assist our efforts, Cure SMA through support from the SMA Industry Collaboration launched SMArt Moves in December 2018. Central to this campaign is a new and easy to use website, where parents may improve their understanding of the early signs of motor delays, watch instructional videos and download helpful checklists to share with their doctor and help address their concerns. Along with take-action resources for parents, there is a special section for healthcare professionals detailing current diagnostic criteria, educational resources, as well as the latest treatment options and protocols.

Our hope is to ensure that all infants and children born with SMA receive life-changing—and in most cases, life-saving—treatment. Today, young children diagnosed with SMA now have a fighting chance – a dramatically improved prognosis and quality of life thanks to a new, effective therapy and other promising treatments on the horizon. The key is early diagnosis and early treatment. Join us at to learn more about the campaign and the resources available for parents, families, and health care providers!

Poster Presentations

Lisa Belter presented two posters at AAN. The first poster was titled “Work Productivity Activity Impairment Results from the Cure SMA 2018 Community Update Survey.” The Work Productivity Activity Impairment (WPAI) is a 7-item questionnaire that measures the work productivity lost due to a specific health problem, such as SMA, over the last 7 days. Productivity is measured by four scores: work time missed (absenteeism); reduced productivity while at work (presenteeism); overall work impairment and reduced productivity while doing regular daily activities (activity impairment). The results are calculated into a percentage that measures to what percent someone’s productivity is affected, both at work and through regular daily activities; a score of 0% represents no impact at all and 100% represents complete impairment. The WPAI was included in the 2018 Cure SMA Community Update Survey. Resuts:169 surveys were completed, and 92 of the surveys were completed by a parent of a child with SMA. On average, parents of children of all SMA types, reported 15.4% absenteeism, 38.3% presenteeism, 43.9% work productivity loss and 51.9% activity impairment due to SMA in the last 7 days. These results show that loss of productivity at work and during regular activities is substantial among caregivers of SMA children.

The second poster presented was titled, “Ambulation Status, Role Participation and Caregiver Assistance among individuals with Spinal Muscular Atrophy Type III: Results from the 2018 Cure SMA Membership Survey.” The purpose of this poster was to enhance understanding of disease burden among children and adults with SMA type III by describing ambulation status, engagement in age-appropriate roles (school, employment), and reliance on caregivers. SMA type was self or caregiver reported. Type III was subtyped as type IIIa (i.e., symptom onset3 years of age; n=39). The results showed that approximately half of the individuals with type IIIa and IIIb had lost their ability to walk independently by the time of the survey. Furthermore, most school-aged children were currently in school, but only about half of the adult participants (n=43) were in school or employed at the time of the survey. Lastly, nearly all patients with type IIIa (89.0%) and the majority with type IIIb (75.0%) reported having a caregiver. These results highlight the disease burden experienced with those with type III SMA.

Jackie Glascock presented a poster titled, “Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening”. The identification of SMA affected infants via newborn screening presents an unprecedented opportunity for achievement of maximal therapeutic benefit through the administration of treatment pre-symptomatically. The treatment algorithm was developed by working group comprised of 15 SMA experts using a modified Delphi process and moderated by a neutral third-party expert. Consensus was reached that SMA infants presymptomatically identified through NBS with 1, 2, or 3 copies of SMN2, should be treated immediately. Consensus was not reached on whether infants with 4 copies of SMN2 identified by NBS should be treated immediately. For those infants with 4 or more copies of SMN2 in whom treated is not initiated immediately, guidelines were developed to monitor for symptom onset which would prompt treatment initiation.

*The SMA-Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The collaboration works together to address scientific, clinical and regulatory topics that are critical to advancing drug development in SMA and will benefit the broader SMA community partners. The SMA-IC is currently comprised of our partners from Astellas, AveXis, Biogen, Cytokinetics, Genentech/Roche, Novartis, and Scholar Rock.

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