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Abstract Details

The Canadian Neuromuscular Disease Registry: A national spinal muscular atrophy (SMA) registry for real world evidence
Neuromuscular and Clinical Neurophysiology (EMG)
S12 - Interventional/Observational Studies of Neuromuscular Diseases (4:08 PM-4:16 PM)
002

The first clinically available disease-modifying therapy for SMA, nusinersen, showed efficacy in randomized controlled trials (RCTs) that had a narrow range of eligibility criteria, limiting the external validity of their findings. Real-world effectiveness must be assessed when therapies are incorporated into general practice across a broader range of patients with multiple comorbidities, variable disease severity, and over a longer timeframe.
To describe real-world evidence from Canadians with Spinal Muscular Atrophy.

The Canadian Neuromuscular Disease Registry (CNDR) is a nationwide pan-neuromuscular disease registry that prospectively collects SMA-specific data in 31 clinics (15 pediatric and 16 adult) across Canada. The objective of the study is to describe real-world evidence from the Canadian Neuromuscular Disease SMA Registry.

We report longitudinal data including motor outcome assessments (CHOP-INTEND, HFMSE, RULM, 6MWT) from 85 patients (26 type I; 32 type II; 23 type III, 3 type IV) between 2015-2019. 

 

Nusinersen was used in 85, 63, 45 and 0% of type I, II, III, and IV respectively. The median age of therapy initiation was 11.3, 49.2, and 115.4 months for type I, II, and III respectively. 

 

Longitudinal analyses of motor outcome measures demonstrate positive change for type I, II, and III SMA patients on therapy.

 

Additional data including use of interventions, comorbidities and hospitalizations from both pediatric and adult patients regardless of therapeutic status are presented. 

The CNDR captures a comprehensive SMA dataset to evaluate long-term real-world experience with available and emerging therapies. 
Authors/Disclosures
Maryam Oskoui, MD, FAAN (Montreal Children's Hospital - McGill University Health Centre)
PRESENTER 		Dr. Oskoui has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for the Association des Neurologues du Quebec. The institution of Dr. Oskoui has received research support from Roche Genetech. The institution of Dr. Oskoui has received research support from Muscular Dystrophy Canada. The institution of Dr. Oskoui has received research support from Canadian Institutes of Health Research. The institution of Dr. Oskoui has received research support from Santhera. Dr. Oskoui has a non-compensated relationship as a Member of the Medical and Scientific Advisory Committee with Muscular Dystrophy Canada that is relevant to AAN interests or activities.
Victoria Hodgkinson No disclosure on file
Bernard Brais, MD Dr. Brais has nothing to disclose.
Craig G. Campbell, MD Dr. Campbell has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Campbell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid. The institution of Dr. Campbell has received research support from Biogen. The institution of Dr. Campbell has received research support from Genzyme. The institution of Dr. Campbell has received research support from Acceleron, AMO, Biogen, Sarepta, Wave, PTC, Pfizer, Roche.
No disclosure on file
No disclosure on file
Hugh J. McMillan, MD (Children's Hospital of Eastern Ontario) Dr. McMillan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis Gene Therapies. Dr. McMillan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis Gene Therapies. The institution of Dr. McMillan has received research support from Roche.
Jiri Vajsar, MD Dr. Vajsar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Vajsar has received research support from CSL Behring.
Lawrence Korngut, MD Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe. Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche.