Abstract Details

Title A Description of Physical Therapy Services Received by Patients Diagnosed with Spinal Muscular Atrophy (SMA)

Topic Anterior Horn

Presentation(s) P03 - (-)

Poster/Presentation
Number 050

Objective

Background BACKGROUND: The standard of care document for SMA recommends and emphasizes the importance of therapy services. There is no known literature discussing the type and frequency of therapy services received and its impact on the natural history of SMA. Two case reports described functional changes over time for patients involved in physical and aquatic therapy programs.

Design/Methods DESIGN/METHODS: We conducted a survey of patients with SMA followed in the Pediatric Neuromuscular Clinical Research Network. Clinical information included age, SMA type, current mobility status, and questions related to therapy services received in the past year or since their last clinic visit , specifically setting, frequency, and type of physical therapy.

Results RESULTS: One-hundred four surveys were completed from October 2011 to September 2012. Twenty-four percent were diagnosed with SMA type 1, 43% with type 2, 32% with type 3, with a mean age of 11.81 (SD 12.11, range 0.02-49.22). Out of the 104 surveys, 64% received physical therapy services (62% in the neuromuscular clinic, 44% at school, 35% at home, and 17% in an outpatient clinic). The duration and frequency of these services varied. The type of therapy our patients received included stretching (68%), strengthening (56%) and aerobic (6%) exercises, developmental activities (30%), aquatic therapy (17%), and standing (23%).

Conclusions CONCLUSIONS: This is the first survey that provides information on the setting, frequency, and type of therapy services received by patients with SMA. Interestingly, 36% of patients do not receive any therapy services despite recommendations as the standard of care for SMA. We plan to continue to collect longitudinal data to better understand the impact of therapy services on the natural history of SMA. Knowledge of this information and its potential impact on function could contribute to standardization of clinical trial planning.

Authors/Disclosures
Sally Dunaway, DPT (Stanford University) PRESENTER	Dr. Dunaway has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Dunaway has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche/Genentech. Dr. Dunaway has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Scholar Rock. Dr. Dunaway has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cure SMA. The institution of Dr. Dunaway has received research support from Cure SMA. The institution of Dr. Dunaway has received research support from Scholar Rock. The institution of Dr. Dunaway has received research support from Ionis Pharmaceuticals.
	No disclosure on file
Jacqueline Montes, PT, EdD, NCS (Columbia University Medical Center)	Ms. Montes has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Ms. Montes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for F. Hoffman LaRoche. Ms. Montes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Ms. Montes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. The institution of Ms. Montes has received research support from NIH/NICHD. The institution of Ms. Montes has received research support from Muscular Dystrophy Association. The institution of Ms. Montes has received research support from Cure SMA.
Allan M. Glanzman, PT (The Children'S Hospital of Philadelphia)	Mr. Glanzman has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Mr. Glanzman has received intellectual property interests from a discovery or technology relating to health care.
	No disclosure on file
	No disclosure on file
William Martens (University of Rochester)	William Martens has nothing to disclose.
Nicole LaMarca	Ms. LaMarca has received personal compensation for serving as an employee of Novartis Gene Therapies.
Marika Hohol, MD (St. Michaels Hospital)	Dr. Hohol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Hohol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Hohol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Genzyme. Dr. Hohol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for EMD Seronon. Dr. Hohol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Hohol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Hohol has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Dr. Hohol has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for EMD Serono.
	No disclosure on file
	No disclosure on file
Douglas M. Sproule, MD (MLBioSolutions)	Dr. Sproule has received personal compensation for serving as an employee of Bridge Bio. Dr. Sproule has received personal compensation for serving as an employee of Novartis, Inc. Dr. Sproule has received stock or an ownership interest from Bridge Bio.
Claudia A. Chiriboga, MD, FAAN (Columbia University)	Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentec. Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Dr. Chiriboga has received research support from Roche. The institution of Dr. Chiriboga has received research support from Avexis/Novartis. The institution of Dr. Chiriboga has received research support from Biogen. The institution of Dr. Chiriboga has received research support from NIH. The institution of Dr. Chiriboga has received research support from Biohaven. The institution of Dr. Chiriboga has received research support from Genentec. Dr. Chiriboga has received publishing royalties from a publication relating to health care.
Richard S. Finkel, MD, FAAN (St. Jude Children's Research Hospital)	Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Capricor. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ReveraGen. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. The institution of Dr. Finkel has received research support from AveXis. The institution of Dr. Finkel has received research support from Biogen. The institution of Dr. Finkel has received research support from Capricor. The institution of Dr. Finkel has received research support from Catabasis. The institution of Dr. Finkel has received research support from ReveraGen. The institution of Dr. Finkel has received research support from Roche. The institution of Dr. Finkel has received research support from Scholar Rock. Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. Dr. Finkel has received personal compensation in the range of $0-$499 for serving as a Speaker in workshop with National Academy of Sciences. Dr. Finkel has a non-compensated relationship as a advisor with n-Lorem Foundation that is relevant to AAN interests or activities. Dr. Finkel has a non-compensated relationship as a Board Member with EveryLife Foundation that is relevant to AAN interests or activities.
Gihan Tennekoon, MD (Children's Hosp of Philadelphi)	No disclosure on file
Basil T. Darras, MD (Children'S Hosp Boston Harvard Med School)	The institution of Dr. Darras has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Darras has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus. The institution of Dr. Darras has received research support from National Institutes of Health/National Institute of Neurological Disorders and Stroke,. The institution of Dr. Darras has received research support from Slaney Family Fund for SMA. The institution of Dr. Darras has received research support from Spinal Muscular Atrophy Foundation. The institution of Dr. Darras has received research support from CureSMA. The institution of Dr. Darras has received research support from Working on Walking Fund . The institution of Dr. Darras has received research support from CHERISH, CS2/CS12 . The institution of Dr. Darras has received research support from Biogen for CS11. The institution of Dr. Darras has received research support from AveXis. The institution of Dr. Darras has received research support from Sarepta Pharmaceuticals. The institution of Dr. Darras has received research support from PTC Therapeutics. The institution of Dr. Darras has received research support from Roche. The institution of Dr. Darras has received research support from Santhera. The institution of Dr. Darras has received research support from Scholar Rock. The institution of Dr. Darras has received research support from Fibrogen. The institution of Dr. Darras has received research support from Summit. Dr. Darras has received publishing royalties from a publication relating to health care. Dr. Darras has received publishing royalties from a publication relating to health care.
Darryl C. De Vivo, MD, FAAN (Columbia University)	Dr. De Vivo has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen and Novartis. Dr. De Vivo has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aspa Therapeutics.
Shree Pandya, DPT	No disclosure on file

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