Full Text Journal Articles by
Author Kathryn Selby


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Association of outcomes in acute flaccid myelitis with identification of enterovirus at presentation: a Canadian, nationwide, longitudinal study.

Carmen Yea, Ari Bitnun, Helen M Branson, Beyza Ciftci-Kavaklioglu, Mubeen F Rafay, Olivier Fortin, Paola Moresoli, Guillaume Sébire, Myriam Srour, Hélène Decaluwe, Louis Marois, Félixe Pelletier, Michelle Barton, Maryam Nabavi Nouri, Jason Brophy, Sunita Venkateswaran, Daniela Pohl, Kathryn Selby, Kevin Jones, Joan Robinson, Aleksandra Mineyko, Christoph Licht, Birgit Ertl-Wagner, E Ann Yeh,

BACKGROUND:Acute flaccid myelitis (AFM) is characterised by rapid onset of limb weakness with spinal cord grey-matter abnormalities on MRI scan. We aimed to assess whether detection of enterovirus in respiratory or other specimens can help predict prognosis in children with AFM. METHODS:In this nationwide, longitudinal study, we evaluated the significance ... Read more >>

Lancet Child Adolesc Health (The Lancet. Child & adolescent health)
[2020, 4(11):828-836]

Cited: 0 times

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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.

Craig Campbell, Richard J Barohn, Enrico Bertini, Brigitte Chabrol, Giacomo Pietro Comi, Basil T Darras, Richard S Finkel, Kevin M Flanigan, Nathalie Goemans, Susan T Iannaccone, Kristi J Jones, Janbernd Kirschner, Jean K Mah, Katherine D Mathews, Craig M McDonald, Eugenio Mercuri, Yoram Nevo, Yann Péréon, J Ben Renfroe, Monique M Ryan, Jacinda B Sampson, Ulrike Schara, Thomas Sejersen, Kathryn Selby, Már Tulinius, Juan J Vílchez, Thomas Voit, Lee-Jen Wei, Brenda L Wong, Gary Elfring, Marcio Souza, Joseph McIntosh, Panayiota Trifillis, Stuart W Peltz, Francesco Muntoni, , , ,

Aim: Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Materials & methods: Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance ... Read more >>

J Comp Eff Res (Journal of comparative effectiveness research)
[2020, 9(14):973-984]

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High prevalence of plasma lipid abnormalities in human and canine Duchenne and Becker muscular dystrophies depicts a new type of primary genetic dyslipidemia.

Zoe White, Chady H Hakim, Marine Theret, N Nora Yang, Fabio Rossi, Dan Cox, Gordon A Francis, Volker Straub, Kathryn Selby, Constadina Panagiotopoulos, Dongsheng Duan, Pascal Bernatchez,

BACKGROUND:Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are allelic X-linked recessive muscle diseases caused by mutations in the DMD gene, with DMD being the more severe form. We have recently shown that increased plasma low-density lipoprotein-associated cholesterol causes severe muscle wasting in the mdx mouse, a mild DMD ... Read more >>

J Clin Lipidol (Journal of clinical lipidology)
[2020, 14(4):459-469.e0]

Cited: 0 times

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De novo pathogenic DNM1L variant in a patient diagnosed with atypical hereditary sensory and autonomic neuropathy.

Maja Tarailo-Graovac, Farah R Zahir, Irena Zivkovic, Michelle Moksa, Kathryn Selby, Sunita Sinha, Corey Nislow, Sylvia G Stockler-Ipsiroglu, Ruth Sheffer, Ann Saada-Reisch, Jan M Friedman, Clara D M van Karnebeek, Gabriella A Horvath,

BACKGROUND:Profiling the entire genome at base pair resolution in a single test offers novel insights into disease by means of dissection of genetic contributors to phenotypic features. METHODS:We performed genome sequencing for a patient who presented with atypical hereditary sensory and autonomic neuropathy, severe epileptic encephalopathy, global developmental delay, and ... Read more >>

Mol Genet Genomic Med (Molecular genetics & genomic medicine)
[2019, 7(10):e00961]

Cited: 2 times

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The clinical spectrum of the congenital myasthenic syndrome resulting from COL13A1 mutations.

Pedro M Rodríguez Cruz, Judith Cossins, Eduardo de Paula Estephan, Francina Munell, Kathryn Selby, Michio Hirano, Reza Maroofin, Mohammad Yahya Vahidi Mehrjardi, Gabriel Chow, Aisling Carr, Adnan Manzur, Stephanie Robb, Pinki Munot, Wei Wei Liu, Siddharth Banka, Harry Fraser, Christian De Goede, Edmar Zanoteli, Umbertina Conti Reed, Abigail Sage, Margarida Gratacos, Alfons Macaya, Marina Dusl, Jan Senderek, Ana Töpf, Monika Hofer, Ravi Knight, Sithara Ramdas, Sandeep Jayawant, Hans Lochmüller, Jacqueline Palace, David Beeson,

Next generation sequencing techniques were recently used to show mutations in COL13A1 cause synaptic basal lamina-associated congenital myasthenic syndrome type 19. Animal studies showed COL13A1, a synaptic extracellular-matrix protein, is involved in the formation and maintenance of the neuromuscular synapse that appears independent of the Agrin-LRP4-MuSK-DOK7 acetylcholine receptor clustering pathway. ... Read more >>

Brain (Brain : a journal of neurology)
[2019, 142(6):1547-1560]

Cited: 2 times

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Abnormal fatty acid metabolism is a core component of spinal muscular atrophy.

Marc-Olivier Deguise, Giovanni Baranello, Chiara Mastella, Ariane Beauvais, Jean Michaud, Alessandro Leone, Ramona De Amicis, Alberto Battezzati, Christopher Dunham, Kathryn Selby, Jodi Warman Chardon, Hugh J McMillan, Yu-Ting Huang, Natalie L Courtney, Alannah J Mole, Sabrina Kubinski, Peter Claus, Lyndsay M Murray, Melissa Bowerman, Thomas H Gillingwater, Simona Bertoli, Simon H Parson, Rashmi Kothary,

OBJECTIVE:Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder leading to paralysis and subsequent death in young children. Initially considered a motor neuron disease, extra-neuronal involvement is increasingly recognized. The primary goal of this study was to investigate alterations in lipid metabolism in SMA patients and mouse models of the ... Read more >>

Ann Clin Transl Neurol (Annals of clinical and translational neurology)
[2019, 6(8):1519-1532]

Cited: 5 times

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Aspartylglycosamine is a biomarker for NGLY1-CDDG, a congenital disorder of deglycosylation.

Hanneke A Haijes, Monique G M de Sain-van der Velden, Hubertus C M T Prinsen, Anke P Willems, Maria van der Ham, Johan Gerrits, Madeline H Couse, Jan M Friedman, Clara D M van Karnebeek, Kathryn A Selby, Peter M van Hasselt, Nanda M Verhoeven-Duif, Judith J M Jans,

BACKGROUND:NGLY1-CDDG is a congenital disorder of deglycosylation caused by a defective peptide:N-glycanase (PNG). To date, all but one of the reported patients have been diagnosed through whole-exome or whole-genome sequencing, as no biochemical marker was available to identify this disease in patients. Recently, a potential urinary biomarker was reported, but ... Read more >>

Mol Genet Metab (Molecular genetics and metabolism)
[2019, 127(4):368-372]

Cited: 2 times

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Diagnostic Yield and Treatment Impact of Targeted Exome Sequencing in Early-Onset Epilepsy.

Michelle Demos, Ilaria Guella, Conrado DeGuzman, Marna B McKenzie, Sarah E Buerki, Daniel M Evans, Eric B Toyota, Cyrus Boelman, Linda L Huh, Anita Datta, Aspasia Michoulas, Kathryn Selby, Bruce H Bjornson, Gabriella Horvath, Elena Lopez-Rangel, Clara D M van Karnebeek, Ramona Salvarinova, Erin Slade, Patrice Eydoux, Shelin Adam, Margot I Van Allen, Tanya N Nelson, Corneliu Bolbocean, Mary B Connolly, Matthew J Farrer,

Targeted whole-exome sequencing (WES) is a powerful diagnostic tool for a broad spectrum of heterogeneous neurological disorders. Here, we aim to examine the impact on diagnosis, treatment and cost with early use of targeted WES in early-onset epilepsy. WES was performed on 180 patients with early-onset epilepsy (≤5 years) of ... Read more >>

Front Neurol (Frontiers in neurology)
[2019, 10:434]

Cited: 4 times

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The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy.

Mathula Thangarajh, Gary L Elfring, Panayiota Trifillis, Joseph McIntosh, Stuart W Peltz, ,

OBJECTIVE:To evaluate the relationship between deficit in digit span and genotype in nonsense mutation (nm) Duchenne muscular dystrophy (DMD) (nmDMD). METHODS:We investigated the relationship between normalized digit-span forward (d-sf) and digit-span backward (d-sb) scores to the location of nmDMD mutations in 169 participants ≥5 to ≤20 years who participated in ... Read more >>

Neurology (Neurology)
[2018, 91(13):e1215-e1219]

Cited: 2 times

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The Canadian Neuromuscular Disease Registry: Connecting patients to national and international research opportunities.

Yi Wei, Anna McCormick, Alex MacKenzie, Erin O'Ferrall, Shannon Venance, Jean K Mah, Kathryn Selby, Hugh J McMillan, Garth Smith, Maryam Oskoui, Gillian Hogan, Laura McAdam, Gracia Mabaya, Victoria Hodgkinson, Josh Lounsberry, Lawrence Korngut, Craig Campbell,

Introduction:Patient registries serve an important role in rare disease research, particularly for the recruitment and planning of clinical trials. The Canadian Neuromuscular Disease Registry was established with the primary objective of improving the future for neuromuscular (NM) patients through the enablement and support of research into potential treatments. Methods:In this ... Read more >>

Paediatr Child Health (Paediatrics & child health)
[2018, 23(1):20-26]

Cited: 2 times

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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

Richard S Finkel, Eugenio Mercuri, Basil T Darras, Anne M Connolly, Nancy L Kuntz, Janbernd Kirschner, Claudia A Chiriboga, Kayoko Saito, Laurent Servais, Eduardo Tizzano, Haluk Topaloglu, Már Tulinius, Jacqueline Montes, Allan M Glanzman, Kathie Bishop, Z John Zhong, Sarah Gheuens, C Frank Bennett, Eugene Schneider, Wildon Farwell, Darryl C De Vivo, ,

BACKGROUND:Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre-messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein. METHODS:We conducted a randomized, ... Read more >>

N Engl J Med (The New England journal of medicine)
[2017, 377(18):1723-1732]

Cited: 254 times

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A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy.

Ronald G Victor, H Lee Sweeney, Richard Finkel, Craig M McDonald, Barry Byrne, Michelle Eagle, Nathalie Goemans, Krista Vandenborne, Alberto L Dubrovsky, Haluk Topaloglu, M Carrie Miceli, Pat Furlong, John Landry, Robert Elashoff, David Cox, ,

OBJECTIVE:To conduct a randomized trial to test the primary hypothesis that once-daily tadalafil, administered orally for 48 weeks, lessens the decline in ambulatory ability in boys with Duchenne muscular dystrophy (DMD). METHODS:Three hundred thirty-one participants with DMD 7 to 14 years of age taking glucocorticoids were randomized to tadalafil 0.3 ... Read more >>

Neurology (Neurology)
[2017, 89(17):1811-1820]

Cited: 16 times

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Compound heterozygous TRPV4 mutations in two siblings with a complex phenotype including severe intellectual disability and neuropathy.

My Linh Thibodeau, Colin H Peters, Katelin N Townsend, Yaoqing Shen, Glenda Hendson, Shelin Adam, Kathryn Selby, Patrick M Macleod, Cynthia Gershome, Peter Ruben, Steven J M Jones, , Jan M Friedman, William T Gibson, Gabriella A Horvath,

TRPV4 encodes a polymodal calcium-permeable plasma membrane channel. Dominant pathogenic mutations in TRPV4 lead to a wide spectrum of abnormal phenotypes. This is the first report of biallelic TRPV4 mutations and we describe two compound heterozygous siblings presenting with a complex phenotype including severe neuromuscular involvement. In light of previously ... Read more >>

Am. J. Med. Genet. A (American journal of medical genetics. Part A)
[2017, 173(11):3087-3092]

Cited: 3 times

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Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.

Craig M McDonald, Craig Campbell, Ricardo Erazo Torricelli, Richard S Finkel, Kevin M Flanigan, Nathalie Goemans, Peter Heydemann, Anna Kaminska, Janbernd Kirschner, Francesco Muntoni, Andrés Nascimento Osorio, Ulrike Schara, Thomas Sejersen, Perry B Shieh, H Lee Sweeney, Haluk Topaloglu, Már Tulinius, Juan J Vilchez, Thomas Voit, Brenda Wong, Gary Elfring, Hans Kroger, Xiaohui Luo, Joseph McIntosh, Tuyen Ong, Peter Riebling, Marcio Souza, Robert J Spiegel, Stuart W Peltz, Eugenio Mercuri, , ,

<label>BACKGROUND</label>Duchenne muscular dystrophy (DMD) is a severe, progressive, and rare neuromuscular, X-linked recessive disease. Dystrophin deficiency is the underlying cause of disease; therefore, mutation-specific therapies aimed at restoring dystrophin protein production are being explored. We aimed to assess the efficacy and safety of ataluren in ambulatory boys with nonsense mutation ... Read more >>

Lancet (Lancet (London, England))
[2017, 390(10101):1489-1498]

Cited: 70 times

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Diagnostic Yield and Treatment Impact of Targeted Exome Sequencing in Early-onset Epilepsy

Michelle Demos, Ilaria Guella, Marna McKenzie, Sarah Buerki, Daniel Evans, Eric Toyota, Cyrus Boelman, Linda Huh, Anita Datta, Aspasia Michoulas, Kathryn Selby, Bruce Bjornson, Gabriella Horvath, Elena Lopez-Rangel, Clara DM van Karnebeek, Ramona Salvarinova, Erin Slade, Patrice Eydoux, Shelin Adam, Margot. Van Allen, Tanya Nelson, Corneliu Bolbocean, Mary Connolly, Matthew Farrer,

Abstract Background To examine the impact on diagnosis, treatment and cost with early use of targeted whole-exome sequencing (WES) in early-onset epilepsy. Methods WES was performed on 50 patients with early-onset epilepsy (≤ 5 years) of unknown cause. Patients were classified as retrospective (epilepsy diagnosis > 6 months) or prospective ... Read more >>

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Cited: 1 time

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Opsoclonus-Myoclonus Syndrome: A New Era of Improved Prognosis?

Armine Galstyan, Colin Wilbur, Kathryn Selby, Juliette Hukin,

Opsoclonus-myoclonus syndrome is an autoimmune neurological disorder characterized by opsoclonus, myoclonus, ataxia, and behavioral changes. Although long-term outcomes have historically been poor, including motor and cognitive disabilities, the advent of new and more aggressive immunotherapy regimens may be improving prognosis in opsoclonus-myoclonus syndrome.We retrospectively reviewed the records of all children diagnosed ... Read more >>

Pediatr Neurol (Pediatric neurology)
[2017, 72:65-69]

Cited: 3 times

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Improved strength on 5-hydroxytryptophan and carbidopa in spinal cord atrophy.

Gabriella A Horvath, Lorelyn Meisner, Kathryn Selby, Robert Stowe, Bruce Carleton,

There is ample evidence of an important role of descending serotonergic projections in modulating spinal motor neuron activation and firing, and experimental studies suggest that 5-HT receptor stimulation can improve motor function after spinal cord injury; however, relevant clinical data is sorely lacking. We describe two sisters with hemiplegic migraine, ... Read more >>

J Neurol Sci (Journal of the neurological sciences)
[2017, 378:59-62]

Cited: 0 times

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Longitudinal Outcomes in the 2014 Acute Flaccid Paralysis Cluster in Canada.

Carmen Yea, Ari Bitnun, Joan Robinson, Aleksandra Mineyko, Michelle Barton, Jean K Mah, Jiri Vajsar, Susan Richardson, Christoph Licht, Jason Brophy, Megan Crone, Shalini Desai, Juliette Hukin, Kevin Jones, Katherine Muir, Jeffrey M Pernica, Robert Pless, Daniela Pohl, Mubeen F Rafay, Kathryn Selby, Sunita Venkateswaran, Geneviève Bernard, E Ann Yeh,

We describe the presenting features and long-term outcome of an unusual cluster of pediatric acute flaccid paralysis cases that occurred in Canada during the 2014 enterovirus D68 outbreak. Children (n = 25; median age 7.8 years) presenting to Canadian centers between July 1 and October 31, 2014, and who met ... Read more >>

J. Child Neurol. (Journal of child neurology)
[2017, 32(3):301-307]

Cited: 13 times

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Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial.

Craig Campbell, Hugh J McMillan, Jean K Mah, Mark Tarnopolsky, Kathryn Selby, Ty McClure, Dawn M Wilson, Matthew L Sherman, Diana Escolar, Kenneth M Attie,

ACE-031 is a fusion protein of activin receptor type IIB and IgG1-Fc, which binds myostatin and related ligands. It aims to disrupt the inhibitory effect on muscle development and provide potential therapy for myopathies like Duchenne muscular dystrophy (DMD).ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a ... Read more >>

Muscle Nerve (Muscle & nerve)
[2017, 55(4):458-464]

Cited: 45 times

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Exome Sequencing and the Management of Neurometabolic Disorders.

Maja Tarailo-Graovac, Casper Shyr, Colin J Ross, Gabriella A Horvath, Ramona Salvarinova, Xin C Ye, Lin-Hua Zhang, Amit P Bhavsar, Jessica J Y Lee, Britt I Drögemöller, Mena Abdelsayed, Majid Alfadhel, Linlea Armstrong, Matthias R Baumgartner, Patricie Burda, Mary B Connolly, Jessie Cameron, Michelle Demos, Tammie Dewan, Janis Dionne, A Mark Evans, Jan M Friedman, Ian Garber, Suzanne Lewis, Jiqiang Ling, Rupasri Mandal, Andre Mattman, Margaret McKinnon, Aspasia Michoulas, Daniel Metzger, Oluseye A Ogunbayo, Bojana Rakic, Jacob Rozmus, Peter Ruben, Bryan Sayson, Saikat Santra, Kirk R Schultz, Kathryn Selby, Paul Shekel, Sandra Sirrs, Cristina Skrypnyk, Andrea Superti-Furga, Stuart E Turvey, Margot I Van Allen, David Wishart, Jiang Wu, John Wu, Dimitrios Zafeiriou, Leo Kluijtmans, Ron A Wevers, Patrice Eydoux, Anna M Lehman, Hilary Vallance, Sylvia Stockler-Ipsiroglu, Graham Sinclair, Wyeth W Wasserman, Clara D van Karnebeek,

Whole-exome sequencing has transformed gene discovery and diagnosis in rare diseases. Translation into disease-modifying treatments is challenging, particularly for intellectual developmental disorder. However, the exception is inborn errors of metabolism, since many of these disorders are responsive to therapy that targets pathophysiological features at the molecular or cellular level.To uncover ... Read more >>

N Engl J Med (The New England journal of medicine)
[2016, 374(23):2246-2255]

Cited: 119 times

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Canadian Paediatric Neurology Workforce Survey and Consensus Statement.

Asif Doja, Serena L Orr, Hugh J McMillan, Adam Kirton, Paula Brna, Michael Esser, Richard Tang-Wai, Philippe Major, Chantal Poulin, Narayan Prasad, Kathryn Selby, Shelly K Weiss, E Ann Yeh, David Ja Callen,

Little knowledge exists on the availability of academic and community paediatric neurology positions. This knowledge is crucial for making workforce decisions. Our study aimed to: 1) obtain information regarding the availability of positions for paediatric neurologists in academic centres; 2) survey paediatric neurology trainees regarding their perceptions of employment issues ... Read more >>

Can J Neurol Sci (The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques)
[2016, 43(3):402-409]

Cited: 2 times

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Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Katharine Bushby, Richard Finkel, Brenda Wong, Richard Barohn, Craig Campbell, Giacomo P Comi, Anne M Connolly, John W Day, Kevin M Flanigan, Nathalie Goemans, Kristi J Jones, Eugenio Mercuri, Ros Quinlivan, James B Renfroe, Barry Russman, Monique M Ryan, Mar Tulinius, Thomas Voit, Steven A Moore, H Lee Sweeney, Richard T Abresch, Kim L Coleman, Michelle Eagle, Julaine Florence, Eduard Gappmaier, Allan M Glanzman, Erik Henricson, Jay Barth, Gary L Elfring, Allen Reha, Robert J Spiegel, Michael W O'donnell, Stuart W Peltz, Craig M Mcdonald, ,

INTRODUCTION:Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of premature stop codons in nonsense mutation (nm) genetic disorders. METHODS:Randomized, double-blind, placebo-controlled study; males ≥ 5 years with nm-dystrophinopathy received study drug orally 3 times daily, ... Read more >>

Muscle Nerve (Muscle & nerve)
[2014, 50(4):477-487]

Cited: 144 times

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Brain MRI abnormalities and spectrum of neurological and clinical findings in three patients with proximal 16p11.2 microduplication.

Isabel Filges, Steven Sparagana, Michael Sargent, Kathryn Selby, Kamilla Schlade-Bartusiak, Gregg T Lueder, Amy Robichaux-Viehoever, Bradley L Schlaggar, Joshua S Shimony, Marwan Shinawi,

The phenotype of recurrent ∼600 kb microdeletion and microduplication on proximal 16p11.2 is characterized by a spectrum of neurodevelopmental impairments including developmental delay and intellectual disability, epilepsy, autism and psychiatric disorders which are all subject to incomplete penetrance and variable expressivity. A variety of brain MRI abnormalities were reported in ... Read more >>

Am J Med Genet A (American journal of medical genetics. Part A)
[2014, 164A(8):2003-2012]

Cited: 4 times

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The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.

Craig M McDonald, Erik K Henricson, R Ted Abresch, Julaine Florence, Michelle Eagle, Eduard Gappmaier, Allan M Glanzman, , Robert Spiegel, Jay Barth, Gary Elfring, Allen Reha, Stuart W Peltz,

INTRODUCTION:An international clinical trial enrolled 174 ambulatory males ≥5 years old with nonsense mutation Duchenne muscular dystrophy (nmDMD). Pretreatment data provide insight into reliability, concurrent validity, and minimal clinically important differences (MCIDs) of the 6-minute walk test (6MWT) and other endpoints. METHODS:Screening and baseline evaluations included the 6-minute walk distance ... Read more >>

Muscle Nerve (Muscle & nerve)
[2013, 48(3):357-368]

Cited: 98 times

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The CNDR: collaborating to translate new therapies for Canadians.

Lawrence Korngut, Craig Campbell, Megan Johnston, Timothy Benstead, Angela Genge, Alex Mackenzie, Anna McCormick, Douglas Biggar, Pierre Bourque, Hannah Briemberg, Colleen O'Connell, Suzan Dojeiji, Joseph Dooley, Ian Grant, Gillian Hogan, Wendy Johnston, Sanjay Kalra, Hans D Katzberg, Jean K Mah, Laura McAdam, Hugh J McMillan, Michel Melanson, Kathryn Selby, Christen Shoesmith, Garth Smith, Shannon L Venance, Joy Wee, ,

BACKGROUND:Patient registries represent an important method of organizing "real world" patient information for clinical and research purposes. Registries can facilitate clinical trial planning and recruitment and are particularly useful in this regard for uncommon and rare diseases. Neuromuscular diseases (NMDs) are individually rare but in aggregate have a significant prevalence. ... Read more >>

Can J Neurol Sci (The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques)
[2013, 40(5):698-704]

Cited: 7 times

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