Medicine and Health Sciences Commons^™

Follow-Up Study Evaluating The Long Term Outcome Of Chondromimetic In The Treatment Of Osteochondral Defects In The Knee, Agnes Berta, Matthew S. Shive, Andrew K. Lynn, Alan Getgood, Saara Totterman, Grahame Busby, Jerome Hollenstein, Gábor Vásárhelyi, Imre Kéki, László Hangody

Bone and Joint Institute

© 2020 by the authors. Scaffolds are thought to be a key element needed for successful cartilage repair treatments, and this prospective extension study aimed to evaluate long-term structural and clinical outcomes following osteochondral defect treatment with a cell-free biphasic scaffold. Structural outcomes were assessed using quantitative 3-D magnetic resonance imaging (MRI) and morphological segmentation to determine the percentage of defect filling and repair cartilage T2 relaxation times, and clinical outcomes were determined with the modified Cincinnati Rating System, and the Knee Injury and Osteoarthritis Outcome Score (KOOS). Seventeen subjects with osteochondral defects in the knee were treated with ChondroMimetic …

Pupil Size Is Modulated By The Size Of Equal-Luminance Gratings, Jie Gao, Athena Ko, Yoshiko Yabe, Melvyn A. Goodale, Juan Chen

Brain and Mind Institute Researchers' Publications

© 2020 The Authors. Pupil size changes with light. For this reason, researchers studying the effect of attention, contextual processing, and arousal on the pupillary response have matched the mean luminance of their stimuli across conditions to eliminate the contribution of differences in light levels. Here, we argue that the match of mean luminance is not enough. In Experiment 1, we presented a circular sinewave grating on a gray background for 2 seconds. The area of the grating could be 3°, 6°, or 9°. The mean luminance of each grating was equal to the luminance of the gray background, such …

Longitudinal Changes Of Brain Microstructure And Function In Nonconcussed Female Rugby Players, Kathryn Y. Manning, Jeffrey S. Brooks, James P. Dickey, Alexandra Harriss, Lisa Fischer, Tatiana Jevremovic, Kevin Blackney, Christy Barreira, Arthur Brown, Robert Bartha, Tim Doherty, Douglas Fraser, Jeff Holmes, Gregory A. Dekaban, Ravi S. Menon

Brain and Mind Institute Researchers' Publications

ObjectiveTo longitudinally assess brain microstructure and function in female varsity athletes participating in contact and noncontact sports.MethodsConcussion-free female rugby players (n = 73) were compared to age-matched (ages 18-23) female swimmers and rowers (n = 31) during the in- and off-season. Diffusion and resting-state fMRI (rs-fMRI) measures were the primary outcomes. The Sports Concussion Assessment Tool and head impact accelerometers were used to monitor symptoms and impacts, respectively.ResultsWe found cross-sectional (contact vs noncontact) and longitudinal (in- vs off-season) changes in white matter diffusion measures and rs-fMRI network connectivity in concussion-free contact athletes relative to noncontact athletes. In particular, mean, axial, …

Total Daily Production And Periodicity Of Melatonin Metabolite In Critically Ill Children, Douglas Fraser

Data and Test Instruments

No abstract provided.

Teaching Neuroimages: Reversible Neuroimaging Findings During Treatment Of Infantile Spasms With Vigabatrin, David Dongkyung Kim, Amit Kumar Sharma, Manas Sharma, Andrea Andrade

Paediatrics Publications

A 10-month-old boy with global developmental delay presented to clinic with a few months of infantile spasms occurring multiple times a day. His seizures continued despite vigabatrin (dosed at 133 mg/kg/d), levetiracetam, and steroid therapy. On vigabatrin, routine follow-up MRI showed abnormal signal change , which may occur in 30.9% of patients.1 Risk is associated with a high peak dose but not cumulative.2 These findings are largely asymptomatic although rarely patients can present with hyperkinetic disorders.2 The imaging findings resolved on 4-month follow-up after tapering vigabatrin. At 18 months of age, the patient continues to have 1 …

Perfusion And Metabolic Neuromonitoring During Ventricular Taps In Infants With Post-Hemorrhagic Ventricular Dilatation., Ajay Rajaram, Lawrence C M Yip, Daniel Milej, Marianne Suwalski, Matthew Kewin, Marcus Lo, Jeffrey J L Carson, Victor Han, Soume Bhattacharya, Mamadou Diop, Sandrine De Ribaupierre, Keith St Lawrence

Paediatrics Publications

Post-hemorrhagic ventricular dilatation (PHVD) is characterized by a build-up of cerebral spinal fluid (CSF) in the ventricles, which increases intracranial pressure and compresses brain tissue. Clinical interventions (i.e., ventricular taps, VT) work to mitigate these complications through CSF drainage; however, the timing of these procedures remains imprecise. This study presents Neonatal NeuroMonitor (NNeMo), a portable optical device that combines broadband near-infrared spectroscopy (B-NIRS) and diffuse correlation spectroscopy (DCS) to provide simultaneous assessments of cerebral blood flow (CBF), tissue saturation (S

Looming And Receding Visual Networks In Awake Marmosets Investigated With Fmri, Justine C. Cléry, David J. Schaeffer, Yuki Hori, Kyle M. Gilbert, Lauren K. Hayrynen, Joseph S. Gati, Ravi S. Menon, Stefan Everling

Brain and Mind Institute Researchers' Publications

© 2020 The Author(s) An object that is looming toward a subject or receding away contains important information for determining if this object is dangerous, beneficial or harmless. This information (motion, direction, identity, time-to-collision, size, velocity) is analyzed by the brain in order to execute the appropriate behavioral responses depending on the context: fleeing, freezing, grasping, eating, exploring. In the current study, we performed ultra-high-field functional MRI (fMRI) at 9.4T in awake marmosets to explore the patterns of brain activation elicited by visual stimuli looming toward or receding away from the monkey. We found that looming and receding visual stimuli …

Reliable And Valid Measures For The Clinical Assessment Of Balance And Gait In Older Adults With Dementia: A Systematic Review, Jacqueline Baker, Danielle De Laat, Emily Kruger, Sarah Mcrae, Sabrina Trung, Carly Zottola, Humberto Omana, Susan W. Hunter

Physical Therapy Publications

No abstract provided.

Agreement Between Original And Rasch-Approved Neck Disability Index, Ze Lu, Joy C. Macdermid, Goris Nazari

Bone and Joint Institute

© 2020 The Author(s). Background: Given the high prevalence of neck pain, the neck disability index (NDI) has been used to evaluate patient status and treatment outcomes. Modified versions were proposed as solutions to measurement deficits in the NDI. However, the original 10-item NDI was scored out of 50 and is still the most frequently administered version. Examining the extent of agreement between traditional and Rasch-based versions using Bland-Altman (B&A) plots will inform our understanding of score differences that might rise from using different versions. Therefore, the objective of current study was to describe the extent of agreement between different …

Consideration Of Pannexin 1 Channels In Covid-19 Pathology And Treatment, Leigh Anne Swayne, Scott R. Johnstone, Chen Seng Ng, Juan C. Sanchez-Arias, Miranda E. Good, Silvia Penuela, Alexander W. Lohman, Abigail G. Wolpe, Victor E. Laubach, X. Michael Koval, Brant E. Isakson

Anatomy and Cell Biology Publications

Copyright © 2020 the American Physiological Society Pannexin 1 (PANX1) is a ubiquitously expressed, channel-forming protein found in a number of tissues throughout the body (e.g., lung, vasculature, liver, central nervous system, immune system) that is important in many key physiological and immune responses (18, 55). PANX1 channels passively flux ATP (predominantly), multiple metabolites, and likely other small anions (37, 39). PANX1 channels regulate inflammation and host responses to several pathogens, including viruses (36, 42, 53). While there is currently no evidence suggesting novel severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and PANX1 directly interact, there is an urgent need …

Screening And Treatment Outcomes In Adults And Children With Type 1 Diabetes And Asymptomatic Celiac Disease: The Cd-Diet Study., Farid H Mahmud, Antoine B M Clarke, Kariym C Joachim, Esther Assor, Charlotte Mcdonald, Fred Saibil, Heather A Lochnan, Zubin Punthakee, Amish Parikh, Andrew Advani, Baiju R Shah, Bruce A Perkins, Caroline S Zuijdwijk, David R Mack, Dror Koltin, Emilia N De Melo, Eugene Hsieh, Geetha Mukerji, Jeremy Gilbert, Kevin Bax, Margaret L Lawson, Maria Cino, Melanie D Beaton, Navaaz A Saloojee, Olivia Lou, Patricia H Gallego, Premysl Bercik, Robyn L Houlden, Ronnie Aronson, Susan E Kirsch, William G Paterson, Margaret A Marcon

Paediatrics Publications

OBJECTIVE: To describe celiac disease (CD) screening rates and glycemic outcomes of a gluten-free diet (GFD) in patients with type 1 diabetes who are asymptomatic for CD.

RESEARCH DESIGN AND METHODS: Asymptomatic patients (8-45 years) were screened for CD. Biopsy-confirmed CD participants were randomized to GFD or gluten-containing diet (GCD) to assess changes in HbA

RESULTS: Adults had higher CD-seropositivity rates than children (6.8% [95% CI 4.9-8.2%,

CONCLUSIONS: CD is frequently observed in asymptomatic patients with type 1 diabetes, and clinical vigilance is warranted with initiation of a GFD.

Are We Missing The Target? Are We Aiming Too Low? What Are The Aerobic Exercise Prescriptions And Their Effects On Markers Of Cardiovascular Health And Systemic Inflammation In Patients With Knee Osteoarthritis? A Systematic Review And Meta-Analysis, Jenna M. Schulz, Trevor B. Birmingham, Hayden F. Atkinson, Emilie Woehrle, Codie A. Primeau, Michael J. Lukacs, Baraa K. Al-Khazraji, Michaela C.M. Khan, Bryn O. Zomar, Robert J. Petrella, Frank Beier, C. Thomas Appleton, J. Kevin Shoemaker, Dianne M. Bryant

Physiology and Pharmacology Publications

© Author(s) (or their employer(s)) 2020. No commercial re-use. See rights and permissions. Published by BMJ. Objectives We systemically reviewed published studies that evaluated aerobic exercise interventions in patients with knee osteoarthritis (OA) to: (1) report the frequency, intensity, type and time (FITT) of exercise prescriptions and (2) quantify the changes in markers of cardiovascular health and systemic inflammation. Data sources PubMed, CINAHL, Scopus; inception to January 2019. Eligibility criteria Randomised clinical trials (RCT), cohort studies, case series. Design We summarised exercise prescriptions for all studies and calculated effect sizes with 95% CIs for between-group (RCTs that compared exercise and …

Appraisal Of Clinical Practice Guideline: Subacromial Decompression Surgery For Adults With Shoulder Pain, Stephanie Reischl, Joy C. Macdermid

Bone and Joint Institute

No abstract provided.

Appraisal Of Clinical Practice Guideline: Optima Revised Recommendations For Non-Pharmacological Management Of Persistent Headaches Associated With Neck Pain, Stephanie Reischl, Armaghan Dabbagh, Joy C. Macdermid

Bone and Joint Institute

No abstract provided.

Music As A Scaffold For Listening To Speech: Better Neural Phase-Locking To Song Than Speech, Christina M. Vanden Bosch Der Nederlanden, Marc F. Joanisse, Jessica A. Grahn

Brain and Mind Institute Researchers' Publications

© 2020 The Authors Neural activity synchronizes with the rhythmic input of many environmental signals, but the capacity of neural activity to entrain to the slow rhythms of speech is particularly important for successful communication. Compared to speech, song has greater rhythmic regularity, a more stable fundamental frequency, discrete pitch movements, and a metrical structure, this may provide a temporal framework that helps listeners neurally track information better than the rhythmically irregular rhythms of speech. The current study used EEG to examine whether entrainment to the syllable rate of linguistic utterances, as indexed by cerebro-acoustic phase coherence, was greater when …

The "Inferior Temporal Numeral Area" Distinguishes Numerals From Other Character Categories During Passive Viewing: A Representational Similarity Analysis, Darren J. Yeo, Courtney Pollack, Rebecca Merkley, Daniel Ansari, Gavin R. Price

Brain and Mind Institute Researchers' Publications

A region in the posterior inferior temporal gyrus (pITG) is thought to be specialized for processing Arabic numerals, but fMRI studies that compared passive viewing of numerals to other character types (e.g., letters and novel characters) have not found evidence of numeral preference in the pITG. However, recent studies showed that the engagement of the pITG is modulated by attention and task contexts, suggesting that passive viewing paradigms may be ill-suited for examining numeral specialization in the pITG. It is possible, however, that even if the strengths of responses to different category types are similar, the distributed response patterns (i.e., …

Expanding The Search For Genetic Biomarkers Of Parkinson's Disease Into The Living Brain, Simon M. Benoit, Hu Xu, Susanne Schmid, Roumiana Alexandrova, Gaganjot Kaur, Bhooma Thiruvahindrapuram, Sergio L. Pereira, Mandar Jog, Matthew O. Hebb

Brain and Mind Institute Researchers' Publications

Altered gene expression related to Parkinson's Disease (PD) has not been described in the living brain, yet this information may support novel discovery pertinent to disease pathophysiology and treatment. This study compared the transcriptome in brain biopsies obtained from living PD and Control patients. To evaluate the novelty of this data, a comprehensive literature review also compared differentially expressed gene (DEGs) identified in the current study with those reported in PD cadaveric brain and peripheral tissues. RNA was extracted from rapidly cryopreserved frontal lobe specimens collected from PD and Control patients undergoing neurosurgical procedures. RNA sequencing (RNA-Seq) was performed and …

Relationship Of Head Circumference And Age In The Prediction Of The Real-Ear-To-Coupler Difference (Recd), Kelli M. Watts, Marlene Bagatto, Sandra Clark-Lewis, Samantha Henderson, Susan Scollie, Judith Blumsack

Communication Sciences and Disorders Publications

Background Pediatric hearing instrument fitting is optimally performed with individually obtained real-ear-to-coupler difference (RECD) measurements. If these measurements cannot be obtained, predicted values based on age are used. Recent evidence obtained from children aged 3-11 years suggests that head circumference (HC) may be a viable alternative or addition to age for use in RECD prediction. Purpose The purpose of the present study was to determine if HC can be used to predict RECDs in infants, children, and adults. Research Design A correlational design was used. HC and RECD values were measured in all participants. Study Sample Participants were 278 North …

Atrx Deletion In Neurons Leads To Sexually Dimorphic Dysregulation Of Mir-137 And Spatial Learning And Memory Deficits., Renee J. Tamming, Vanessa Dumeaux, Yan Jiang, Sarfraz Shafiq, Luana Langlois, Jacob Ellegood, Lily R. Qiu, Jason P. Lerch, Nathalie G. Bérubé

Paediatrics Publications

ATRX gene mutations have been identified in syndromic and non-syndromic intellectual disabilities in humans. ATRX is known to maintain genomic stability in neuroprogenitor cells, but its function in differentiated neurons and memory processes remains largely unresolved. Here, we show that the deletion of neuronal Atrx in mice leads to distinct hippocampal structural defects, fewer presynaptic vesicles, and an enlarged postsynaptic area at CA1 apical dendrite-axon junctions. We identify male-specific impairments in long-term contextual memory and in synaptic gene expression, linked to altered miR-137 levels. We show that ATRX directly binds to the miR-137 locus and that the enrichment of the …

Transient Hyponatremia Of Prematurity Caused By Mild Bartter Syndrome Type Ii: A Case Report., Subhrata Verma, Rahul Chanchlani, Victoria Mok Siu, Guido Filler

Paediatrics Publications

BACKGROUND: Bartter syndrome subtypes are a group of rare renal tubular diseases characterized by impaired salt reabsorption in the tubule, specifically the thick ascending limb of Henle's loop. Clinically, they are characterized by the association of hypokalemic metabolic alkalosis, hypercalciuria, nephrocalcinosis, increased levels of plasma renin and aldosterone, low blood pressure and vascular resistance to angiotensin II. Bartter syndrome type II is caused by mutations in the renal outer medullary potassium channel (ROMK) gene (KCNJ1), can present in the newborn period and typically requires lifelong therapy.

CASE PRESENTATION: We describe a case of a prematurely born female infant presenting with …

Does Endoscopic Sinus Surgery Alter The Biomechanics Of The Orbit?, Leigh J. Sowerby, Matthew S. Harris, Rootu Joshi, Marjorie Johnson, Tom Jenkyn, Corey C. Moore

Bone and Joint Institute

© 2020 The Author(s). Objective: The purpose of this study is to determine if removal of ethmoid cell septations as commonly performed in endoscopic sinus surgery leads to a change in orbital wall fracture patterns and the force required to create them. Methods: Six fresh-frozen cadaveric heads were acquired and underwent endoscopic uncinectomy, maxillary antrostomy, and anterior and posterior ethmoidectomy on one, randomized, side. The contralateral sinuses were used as intra-specimen control. Hyaluronic acid gel globe injections were performed to simulate normal intra-ocular pressure. Post-op CT scans confirmed no orbital fractures or violation of the lamina papyracea prior to trauma …

Effects Of A Postnatal Atrx Conditional Knockout In Neurons On Autism-Like Behaviours In Male And Female Mice., Nicole Martin-Kenny, Nathalie G Bérubé

Paediatrics Publications

BACKGROUND: Alpha-thalassemia/mental retardation, X-linked, or ATRX, is an autism susceptibility gene that encodes a chromatin remodeler. Mutations of ATRX result in the ATR-X intellectual disability syndrome and have been identified in autism spectrum disorder (ASD) patients. The mechanisms by which ATRX mutations lead to autism and autistic-like behaviours are not yet known. To address this question, we generated mice with postnatal Atrx inactivation in excitatory neurons of the forebrain and performed a battery of behavioural assays that assess autistic-like behaviours.

METHODS: Male and female mice with a postnatal conditional ablation of ATRX were generated using the Cre/lox system under the …

Longitudinal Changes Of Brain Microstructure And Function In Non-Concussed Female Rugby Players, Douglas Fraser

Data and Test Instruments

No abstract provided.

Maternal Immune Activation Alters Fetal Brain Development And Enhances Proliferation Of Neural Precursor Cells In Rats, Kelly J. Baines, Dendra M. Hillier, Faraj L. Haddad, Nagalingam Rajakumar, Susanne Schmid, Stephen J. Renaud

Brain and Mind Institute Researchers' Publications

Maternal immune activation (MIA) caused by exposure to pathogens or inflammation during critical periods of neurodevelopment is a major risk factor for behavioral deficits and psychiatric illness in offspring. A spectrum of behavioral abnormalities can be recapitulated in rodents by inducing MIA using the viral mimetic, PolyI:C. Many studies have focused on long-term changes in brain structure and behavioral outcomes in offspring following maternal PolyI:C exposure, but acute changes in prenatal development are not well-characterized. Using RNA-Sequencing, we profiled acute transcriptomic changes in rat conceptuses (decidua along with nascent embryo and placenta) after maternal PolyI:C exposure during early gestation, which …

Association Between Changes In Subjective And Objective Measures Of Mobility In People With Lower Limb Amputations After Inpatient Rehabilitation, Gabrielle Cieslak, Humberto Omana, Edward Madou, Courtney Frengopoulos, Ricardo Viana, Michael W Payne, Susan W. Hunter

Physical Therapy Publications

Functional recovery for people with lower limb amputations is quantified using objective or subjective measures of performance. In this brief report, the prospective relationship between objective and subjective mobility after rehabilitation was evaluated in people with lower limb amputations. Adults undergoing inpatient prosthetic rehabilitation for a first unilateral transtibial or transfemoral level lower limb amputation were recruited. Assessment times: discharge and 4-mo follow-up. Gait velocity and the L Test under single- and dual-task conditions measured objective mobility. The Prosthetic Evaluation Questionnaire (section 4 and question 5b) measured subjective mobility. Paired t tests and Pearson correlation analysis evaluated change over time …

Fri0547 The Effect Of Corrected Inflammation, Oxidative Stress And Endothelial Dysfunction On Fmd Levels In Patients With Selected Chronic Diseases: A Quasi-Experimental Study., Mahmut Ilker Yilmaz, Micol Romano, Mustafa Kemal Basarali, Abdelbaset Elzagallaai, Murat Karaman, Zeynep Demir, Muhammet Fatih Demir, Fatih Akcay, Melik Seyrek, Nuri Haksever, David Piskin, Rolando Cimaz, Michael Rieder, Erkan Demirkaya

Paediatrics Publications

While the pathophysiology of chronic disorders varies there are three basic mechanisms - inflammation, oxidative stress and endothelial dysfunction - that are common in many chronic diseases. However, the failure of these mechanisms to work synchronously can lead to morbidity complicating the course of many chronic diseases. We analyzed data of 178 patients from cohorts with selected chronic diseases in this quasi-experimental study. Endothelial dysfunction was determined by flow-mediated dilatation (FMD) and asymmetric dimethylarginine (ADMA) levels. Serum ADMA, high sensitive C-reactive protein (hs-CRP), serum PTX3, malondialdehyde (MDA), Cu/Zn-superoxide dismutase (Cu/Zn-SOD), glutathione peroxidase (GSH-Px) levels and FMD were studied in baseline …

The Influence Of Depression-Ptsd Comorbidity On Health-Related Quality Of Life In Treatment-Seeking Veterans, Callista Forchuk, Anthony Nazarov, Renee Hunt, Brent Davis, Kate St. Cyr, J Donald Richardson

Psychiatry Publications

Objective: Posttraumatic stress disorder (PTSD) and depression substantially impair healthrelated quality of life (HRQOL) for many Canadian Armed Forces (CAF) veterans. Although PTSD and depression are highly comorbid, little is known about whether the disorders may interact in their association with HRQOL. We sought to investigate whether depressive symptoms modify the relation between PTSD and HRQOL in treatment-seeking veterans. Method: We accessed the clinical data of 545 CAF veterans aged 18 to 65 years who were seeking treatment at a specialized clinic in London, Ontario. We used hierarchical linear regression to assess the additive and multiplicative relations between depression and …

The Effect Of Corrected Inflammation, Oxidative Stress And Endothelial Dysfunction On Fmd Levels In Patients With Selected Chronic Diseases: A Quasi-Experimental Study., Mahmut Ilker Yilmaz, Micol Romano, Mustafa Kemal Basarali, Abdelbaset Elzagallaai, Murat Karaman, Zeynep Demir, Muhammet Fatih Demir, Fatih Akcay, Melik Seyrek, Nuri Haksever, David Piskin, Rolando Cimaz, Michael Rieder, Erkan Demirkaya

Paediatrics Publications

While the pathophysiology of chronic disorders varies there are three basic mechanisms - inflammation, oxidative stress and endothelial dysfunction - that are common in many chronic diseases. However, the failure of these mechanisms to work synchronously can lead to morbidity complicating the course of many chronic diseases. We analyzed data of 178 patients from cohorts with selected chronic diseases in this quasi-experimental study. Endothelial dysfunction was determined by flow-mediated dilatation (FMD) and asymmetric dimethylarginine (ADMA) levels. Serum ADMA, high sensitive C-reactive protein (hs-CRP), serum PTX3, malondialdehyde (MDA), Cu/Zn-superoxide dismutase (Cu/Zn-SOD), glutathione peroxidase (GSH-Px) levels and FMD were studied in baseline …

Prevalence And Clinical Features Of Inflammatory Bowel Diseases Associated With Monogenic Variants, Identified By Whole-Exome Sequencing In 1000 Children At A Single Center, Eileen Crowley, Neil Warner, Jie Pan, Sam Khalouei, Abdul Elkadri, Karoline Fiedler, Justin Foong, Andrei L Turinsky, Dana Bronte-Tinkew, Shiqi Zhang

Paediatrics Publications

BACKGROUND & AIMS: A proportion of infants and young children with inflammatory bowel diseases (IBDs) have subtypes associated with a single gene variant (monogenic IBD). We aimed to determine the prevalence of monogenic disease in a cohort of pediatric patients with IBD.

METHODS: We performed whole-exome sequencing analyses of blood samples from an unselected cohort of 1005 children with IBD, aged 0-18 years (median age at diagnosis, 11.96 years) at a single center in Canada and their family members (2305 samples total). Variants believed to cause IBD were validated using Sanger sequencing. Biopsies from patients were analyzed by immunofluorescence and …

Randomized Phase 2 Trial And Open-Label Extension Of Domagrozumab In Duchenne Muscular Dystrophy., Kathryn R. Wagner, Hoda Z. Abdel-Hamid, Jean K. Mah, Craig Campbell, Michela Guglieri, Francesco Muntoni, Yasuhiro Takeshima, Craig M. Mcdonald, Anna Kostera-Pruszczyk, Peter Karachunski, Russell J. Butterfield, Eugenio Mercuri, Chiara Fiorillo, Enrico S. Bertini, Cuixia Tian, Jeffery Statland, Alesia B. Sadosky, Vivek S. Purohit, Sarah P. Sherlock, Jeffrey P. Palmer, Michael Binks, Lawrence Charnas, Shannon Marraffino, Brenda L. Wong

Paediatrics Publications

We report results from a phase 2, randomized, double-blind, 2-period trial (48 weeks each) of domagrozumab and its open-label extension in patients with Duchenne muscular dystrophy (DMD). Of 120 ambulatory boys (aged 6 to <16 >years) with DMD, 80 were treated with multiple ascending doses (5, 20, and 40 mg/kg) of domagrozumab and 40 treated with placebo. The primary endpoints were safety and mean change in 4-stair climb (4SC) time at week 49. Secondary endpoints included other functional tests, pharmacokinetics, and pharmacodynamics. Mean (SD) age was 8.4 (1.7) and 9.3 (2.3) years in domagrozumab- and placebo-treated patients, respectively. Difference in mean …