LAST_NAME FIRST_NAME Poster # Castiglioni Claudia 1 Belter Lisa 2 Belter Lisa 3 Dabbous Omar 4 Bassano Michela 5 De Amicis Ramona 6 Bertoli Simona 7

Transcription

1 LAST_NAME FIRST_NAME Poster # Castiglioni Claudia 1 Belter Lisa 2 Belter Lisa 3 Dabbous Omar 4 Bassano Michela 5 De Amicis Ramona 6 Bertoli Simona 7 Chen Tai-Heng 8 Mastella Chiara 9 Babb Jaanai 10 Eagen Shelly 11 Rothman Jennifer 12 Goodwin Ashley 13 Crippa Patricia 14 Townsend Elise 15 Dunaway Young Sally 16 Duong Tina 17 Alfano Lindsay 18 Castiglioni Claudia 19 Salazar Rachel 20 LoMauro Antonella 21 Monges Soledad 22 Dixon Stacy 23 Otto Louise 24 Shieh Perry 25 Sumner Charlotte 26 Tizzano Eduardo 27 Bullivant Joanne 28 Bullivant Joanne 29 O'Connor Nicole 30 Zappa Grazia 31 Zappa Grazia 32 LaMarca Nicole 33 Farrar Michelle 34 Farrar Michelle 35 Glascock Jackie 36 Newcomb Tara 37 Swoboda Kathryn 38 Swoboda Kathryn 39 Ascadi Gyula 40 Bharucha Goebel Diana 41 Brady Caitlin 42 Coskery Shelley 43 Elsheikh Bakri 44 Ikuta Ichiro 45 LaMarca Nicole 46

2 Romero Danielle 47 Rao Vamshi 48 Toops Jeannie 49 Kundrat Katherine 50 Cruz Rosangel 51 Cruz Rosangel 52

3 Title CAREGIVER BURDEN IN SPINAL MUSCULAR ATROPHY PARENTS IN CHILE Survival and Ventilation among those with Type I SMA: Results from the 2017 Cure SMA Membership Sur Cure SMA Membership: Findings from the 2018 Membership Survey Economic Burden of Infant-Onset (Type 1) Spinal Muscular Atrophy: A Retrospective Claims Database An Vitamins intake in italian children with spinal muscular atrophy type 1 and 2 Is Fat Free Mass amount associated to protein intake in children with Spinal Muscular Atrophy Type I? Nutritional status and body composition of children with spinal muscular atrophy type 1 before pharmac Hospitalization and Emergency Room Visits among Children with Spinal Muscular Atrophy: Experience of Multidisciplinary Italian Program for Early Parental Empowerment (PEPE) in SMA type 0 and type I (SMA1 Characterization of Infant and Early Childhood Spinal Muscular Atrophy Patients and Their Transitions of Improving Patient Access to Multidisciplinary Clinical Care for Patients with Neuromuscular Disorders A modified physiotherapeutic scoliosis specific exercise program (Schroth method) and night bracing stab Performance of the six minute walk test in pediatric neuromuscular disease: Review and comparison to h Exploratory study for comparison of passive gait training methods in nonabulatory SMA children Supported standing in SMA Types 1 and 2: Head rotation control and respiratory support predict meanin Scoliosis is Negatively Associated with Motor Function in Children with Later-Onset SMA Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Feasibility for More than just fun and games: Can the ACTIVE workspace volume video game quantify meaningful chang Use of inertial sensors in motor function assessment in spinal muscular atrophy type 3. An exploratory st Prospective Quantitative Study of Passive Lower Extremity Range of Motion (ROM) in Spinal Muscular At Impaired ribcage muscles function alters ribcage structure in spinal muscular atrophy (SMA) children Wide spectrum of age at onset and clinical variability in SMA infants and children with 3 SMN2 copies. Diagnosis of 5q-related SMA in an asymptomatic woman after undergoing routine SMN1 carrier screenin MR imaging of muscle as a biomarker for disease progression in SMA Quantitative Determination of SMN2 Copy Number using Next Generation Sequencing and Correlation to Neurofilament as a Potential Biomarker for Spinal Muscular Atrophy: Rationale Based on Animal and Hum Update on SMN1 mutations in Spanish SMA patients: description of new rare stop and splicing mutations Collaborative Data by TREAT-NMD Registries to support post-marketing surveillance in Spinal Muscular A Aligning spinal muscular atrophy registries worldwide for post-marketing surveillance The Updated Family Guide for Spinal Muscular Atrophy Two case reports of spinal muscular atrophy Type II with Autism Spectrum Disorders Cognitive Development (IQ), Language Comprehension and Motor Functioning in SMA1 Children using AA Spinal Muscular Atrophy: Cases of Normal Non-Verbal Receptive Vocabulary and Reasoning Skills in Non- "The whole game is changing and you've got hope": Decision making in Spinal Muscular Atrophy care. Newborn Screening Pilot for Diagnosis of SMA/PID by Multiplex qpcr on Guthrie Card DNA Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening Statewide newborn screening for SMA in Utah, implementation and early results The NBSTRN SPOT SMA Longitudinal Population Database Repository (LPDR) Utilization of the Newborn Screening Translational Research Network (NBSTRN): Project Cure SMA Longit Early therapeutic response to Spinraza in SMA type 3 Implementation and Experiences with nusinersen (Spinraza) for infants and children with Spinal Muscular Spinraza and Inpatient Rehabilitation: What's the relationship? Nusinersen Protocol and Treatment of Patients with Spinal Muscular Atrophy (SMA) The Ohio State University Wexner Medical Center (OSUWMC) experience in treating adults with Spinal M Image-guided Spinraza (nusinersen) injection for patients with spinal muscular atrophy: Taking great care Development of a Nusinersen Treatment Program for Spinal Muscular Atrophy at a Large Academic

4 Value of an advanced practice nurse in care coordination of Spinraza treatment for SMA patients. Challenges and outcomes with nusinersen in a wide spectrum of SMA patients: The Lurie Children's Hosp The FAST (Facilitating Access to SMA Treatment) Program at Nationwide Children's Hospital: a Model for Utilizing SATCo to Assess Trunk Control in Patient's with SMA Undergoing Spinraza Treatment Benefit-Risk Survey Results for Spinal Muscular Atrophy Informing Drug Development in SMA Results of Nationwide Survey to Evaluate SMA Clinical Trial Capacity at Sites with Experience Conducting

5 bilized scoliotic curves and improved pulmonary function in young boy with Spinal Muscle Atrophy Type

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7 3 - A Case Report