GM2 Gangliosidosis: Getting the Most out of Patient Surveys
|
|
- Brook Dorsey
- 5 years ago
- Views:
Transcription
1 GM2 Gangliosidosis: Getting the Most out of Patient Surveys NIH Natural History Workshop May 17, 2012 Florian S. Eichler, MD Massachusetts General Hospital
2 GM2 Gangliosidosis What we know AR inherited lysosomal beta-hexosaminidase deficiency ganglioside accumulation in brain neurodegeneration in children but also adults no effective treatments failed systemic delivery of enzyme promising data on intracranial gene delivery in animals
3 GM2 Gangliosidosis What we know Infantile GM2 Striking pathology but little data on rate of clinical progression
4 GM2 Gangliosidosis The Challenges rate of functional decline unknown factors affecting survival not systematically described heterogeneous clinical spectrum infantile form: most severe and progressive juvenile form: rarer and more variable adult form: chronic, slow biomarkers of neurodegeneration unknown
5 GM2 Gangliosidosis The Challenges literature mostly case reports low prevalence and incidence most patients far advanced by time of diagnosis risks of travel risks of intubation limited ability for large prospective cohort limited resources
6 Begin with the end in mind plan for intracranial AAV-mediated gene delivery significant survival advantage in animal models does the current animal work justify moving towards a human clinical trial? what patient population is the first one to test? is the target of administration appropriate? Is the level of risk justified in light of the known rate of disease progression? what endpoints should be studied?
7 GM2 Gangliosidosis The Opportunities well organized patient advocacy group prospect of intervention motivated families and advocates collaborative team of investigators (young + old) collective memory of the disease rapid disease progression in infants may be most quantifiable and homogeneous
8 The Strategy for Infantile GM2 develop patient surveys to estimate survival and quantify gain and loss of specific developmental milestones identify and recruit patients through advocacy organization collect anonymized surveys (waiver of written consent)
9 The Surveys for Infantile GM2 12 sections, 18 pages 70 yes/no questions followed by requests to time events options to state uncertainty or not applicable validation questions prior to send-out we had test-runs: physicians of different specialties patient advocates family members
10 Bley et al. Pediatrics 2011
11 The Surveys for Infantile GM2 initial skepticism poor first response follow-up s from advocacy group plug at annual meetings visible presence at fundraiser events work to create buy-in 237 families contacted, 97 surveys received (5 HSCT analyzed separately)
12 The Strategy for Infantile GM2 we complemented surveys with life-span data from patients whose families did not respond to surveys literature search dating back to 1881 (>2000 papers)
13 Mortality data according to cohort and birth year for patients with infantile GM2 gangliosidosis n=121 n=103 n=92 Bley et al. Pediatrics 2011
14 Survival curves for patients with infantile GM2 gangliosidosis 47 months Bley et al. Pediatrics 2011
15 Disease progression of infantile GM2 gangliosidosis Bley et al. Pediatrics 2011
16 Bley et al. Pediatrics 2011
17 Bley et al. Pediatrics 2011
18 What did we learn? caregivers can provide a detailed recollection of distinct clinical findings (baby books), but milestones may be tainted by subjective impression and cannot be objectively verified. details on the course of regression assist in choice of outcome measures and design trials for future interventions first draft of disease specific clinical scoring system Bley et al. Pediatrics 2011
19 (max. 10 points)
20 Limitations retrospective nature self-reporting by parents and other family members features harder to isolate (e.g.vocalizing) more prone to misrepresentation cannot exclude recall bias, ascertainment bias and bias of potentially missing data need for prospective validation
21 Biomarkers: Brain MRI Abnormalities in GM2 families also sent advocacy group brain MRIs opportunity to develop MRI scoring system location of abnormalities location of atrophy GM2 control
22 Biomarkers: Brain MRI Scoring System for GM2 (max. 20 points)
23 Biomarkers: Brain MRI Scoring System for GM2 Infantile Juvenile Adult Total MRI score Age at MRI (years) Age at MRI (years) Age at MRI (years) 11.1 points (range 9-15) 4.3 points (range 3-7) 1 point (range 0-2) significant differences between subtypes but no serial data to suggest that MRI tracks progression
24 How does this influence clinical trial design? choice of patient population: most infantile patients are too advanced by time of diagnosis to benefit from intervention include only infantile patients who had gained milestones include juvenile patients that can still walk independently
25 How does this influence clinical trial design? choice of outcome measures: measure retention of clinical function need prospective studies (6MWT, GMFS, etc.) develop biomarkers (MRI, gangliosides in CSF) correlate biomarkers with clinical course
26 Summary first rough sketch of the GM2 landscape surveys chart clinical course MRI scoring system describes anatomic burden many details need to be filled in questions raised and room for growth temporal and spatial benchmarks for future studies much can be learned from retrospective surveys
27 Late Phase Clinical Early Phase Clinical IND Enabling Pathophysiology MR Imaging Trial Design Biomarkers Prospective Studies Literature Patient Surveys
28 Aknowledgements TSGT Consortium National Tay Sachs and Allied Diseases Association NIH UL1 RR NIH K08NS52550 Lysosomal Disease Network 5U54NS MGH: NTSAD: Annette Bley, Paul Caruso Ourania Giannikopoulos Doug Hayden Kim Kubilus, Susan Kahn
PROJECT TRICALS AN INTERNATIONAL COLLABORATION TO FIND EFFECTIVE TREATMENTS FOR AMYOTROPHIC LATERAL SCLEROSIS
PROJECT TRICALS AN INTERNATIONAL COLLABORATION TO FIND EFFECTIVE TREATMENTS FOR AMYOTROPHIC LATERAL SCLEROSIS BACKGROUND Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease (MND) is
More informationWhen a Threshold Crossing approach may and may not be appropriate: A Case Study in SMA
When a Threshold Crossing approach may and may not be appropriate: A Case Study in SMA EFSPI Regulatory Statistics Workshop, 24-25 th September 2018 Carol Reid and Uli Burger picture placeholder Outline
More informationFaculty. W4: Identifying Patients with Rare Disorders Using Administrative Data. O Dan Malone, RPh, PhD University of Arizona
W4: Identifying Patients with Rare Disorders Using Administrative Data Faculty O Dan Malone, RPh, PhD University of Arizona O Danny Yeh, PhD Biogen O Ed Armstrong Strategic Therapeutics, LLC O Eric Bell,
More informationResults. NeuRA Motor dysfunction April 2016
Introduction Subtle deviations in various developmental trajectories during childhood and adolescence may foreshadow the later development of schizophrenia. Studies exploring these deviations (antecedents)
More informationMY.JHSPH.EDU HOME About the Center Projects Faculty and Staff Publications Contact Us PFRH Home Contact Us ADDRESS Johns Hopkins Bloomberg School of Public Health Department of Population, Family and Reproductive
More informationAccelerating Innovation in Statistical Design
Implementing a National Cancer Clinical Trials System for the 21 st Century, Workshop #2 Session #5: Accelerating Innovation Through Effective Partnerships Accelerating Innovation in Statistical Design
More informationHealth Studies 315. Clinical Epidemiology: Evidence of Risk and Harm
Health Studies 315 Clinical Epidemiology: Evidence of Risk and Harm 1 Patients encounter (possibly) risky exposures Alcohol during pregnancy (fetal risk) Electromagnetic fields (cancer risk) Vasectomy
More informationE11(R1) Addendum to E11: Clinical Investigation of Medicinal Products in the Pediatric Population Step4
E11(R1) Addendum to E11: Clinical Investigation of Medicinal Products in the Step4 October 2017 International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use 1 Legal
More informationShould Universal Carrier Screening be Universal?
Should Universal Carrier Screening be Universal? Disclosures Research funding from Natera Mary E Norton MD University of California, San Francisco Antepartum and Intrapartum Management June 15, 2017 Burden
More informationIncreased Density of the Thalamus on CT Scans in Patients with GM2 Gangliosidoses
125 Increased Density of the Thalamus on CT Scans in Patients with GM2 Gangliosidoses Jan Brismar 1 Gudrun Brismar 2 Robert Coates 1 Generoso Gascon 3 Pinar Ozand 3 In 13 patients, the GM2 gangliosidoses,
More informationNCI Cancer Moonshot. Christine Siemon National Cancer Institute, NIH. November 30, 2017
NCI Cancer Moonshot Christine Siemon National Cancer Institute, NIH November 30, 2017 Presidential Memo 2016 Overall Goals of the Cancer Moonshot Accelerate progress in cancer, including prevention & screening
More informationPaediatric HIV -Developmental Aspects. Dr Kirsty Donald Division of Developmental Paediatrics Red Cross Children s Hospital
Paediatric HIV -Developmental Aspects Dr Kirsty Donald Division of Developmental Paediatrics Red Cross Children s Hospital Normal Development Development occurs with sequential acquisition of skills in
More informationBias. A systematic error (caused by the investigator or the subjects) that causes an incorrect (overor under-) estimate of an association.
Bias A systematic error (caused by the investigator or the subjects) that causes an incorrect (overor under-) estimate of an association. Here, random error is small, but systematic errors have led to
More informationWhat the IOM Report Means for Basic and Clinical Research December 1, 2012
What the IOM Report Means for Basic and Clinical Research December 1, 2012 Story C. Landis, PhD Director, National Institute of Neurological Disorders and Stroke American Epilepsy Society Annual Meeting
More informationStandardize and Optimize. Trials and Drug Development
Informatics Infrastructure to Standardize and Optimize Quantitative Imaging in Clinical Trials and Drug Development Daniel L. Rubin, MD, MS Assistant Professor of Radiology Member, Stanford Cancer Center
More informationWorld Health Organization Growth Standards. First Nations and Inuit Health Alberta Region: Training Module May 2011
World Health Organization Growth Standards First Nations and Inuit Health Alberta Region: Training Module May 2011 Acknowledgements First Nation and Inuit Health Alberta Region would like to thank the
More informationThe role of the laboratory in diagnosing lysosomal disorders
The role of the laboratory in diagnosing lysosomal disorders Dr Guy Besley, formerly Willink Biochemical Genetics Unit, Manchester Children s Hospital, Manchester M27 4HA, UK. Lysosomal disorders What
More informationBirth Stress and Self-Reported Sleep Difficulty
Sleep, 8(3):222-226 1985 Raven Press, New York Birth Stress and Self-Reported Sleep Difficulty Stanley Coren and * Alan Searleman Department of Psychology, University of British Columbia, Vancouver, British
More informationDelphi Survey Results. MPIs: Drs. William Dale, Arti Hurria, Supriya Mohile
Delphi Survey Results MPIs: Drs. William Dale, Arti Hurria, Supriya Mohile Cancer and Aging Research Group (CARG) A Delphi Investigation Of Geriatric Oncology Experts Sustainable Infrastructure That Supports
More informationIntegrative Medicine: A Foundation for Prospective Health Care
Integrative Medicine: A Foundation for Prospective Health Care Distinguished Lectures in the Science of Complementary and Alternative Medicine National Institutes of Health April 22, 2005 c. 3,500 BCE
More informationWhat is dementia? What is dementia?
What is dementia? What is dementia? What is dementia? Dementia is an umbrella term for a range of progressive conditions that affect the brain. There are over 200 subtypes of dementia, but the five most
More informationSELECTIVE VULNERABILITY (HYPOXIA AND HYPOGLYCEMIA)
DEFICIENCY OF METABOLITE -HYPOXIA AND HYPOGLYCEMIA -HYPOVITAMINOSIS SELECTIVE VULNERABILITY (HYPOXIA AND HYPOGLYCEMIA) -SPECIFIC CELL TYPE NEURONS>OLIGODENDROCYTES>ASTROCYTES -SPECIFIC BRAIN REGION PYRAMIDAL
More informationImplementing change: An overview of the NSAP Peer Mentorship
NSAP CQI Webinar Implementing change: An overview of the NSAP Peer Mentorship Wednesday 28 th August 2013 Peer Mentorship Don t stress your CQI, Peer Mentors will help you fly Don t stress your CQI, Book
More informationWhat is dementia? What is dementia?
What is dementia? What is dementia? What is dementia? Dementia is an umbrella term for a range of progressive conditions that affect the brain. It has been identified that there are over 200 subtypes of
More informationNew NCCIH Funding Opportunities for Mind and Body Clinical Trials. April 24, 2017
New NCCIH Funding Opportunities for Mind and Body Clinical Trials April 24, 2017 Introductions Wendy Weber, N.D., Ph.D., M.P.H. Branch Chief, Clinical Research in Complementary and Integrative Health Branch,
More informationKevin M. Flanigan, MD Center for Gene Therapy Nationwide Children s Hospital Columbus, OH
A Global Phase 1/2 Clinical Trial of Systemic Gene Transfer of scaav9.u1a.hsgsh for MPS IIIA: Safety, Tolerability, and Efficacy Kevin M. Flanigan, MD Center for Gene Therapy Nationwide Children s Hospital
More informationPACES Research Meeting May 12, Boston, MA. Introduction
PACES Research Meeting May 12, 2015 Boston, MA Introduction On Tuesday May 12 before the annual PACES symposium, a research meeting was held. This was sponsored by Johnson and Johnson. This meeting represents
More informationNIH HEAL Initiative: Program Update and Funding Opportunities for Pain Research Available through NIH
NIH HEAL Initiative: Program Update and Funding Opportunities for Pain Research Available through NIH AAMC Webinar November 8, 2018 Walter J. Koroshetz, MD Director, National Institute of Neurological
More informationHandout 1: Introduction to the Research Process and Study Design STAT 335 Fall 2016
DESIGNING OBSERVATIONAL STUDIES As we have discussed, for the purpose of establishing cause-and-effect relationships, observational studies have a distinct disadvantage in comparison to randomized comparative
More informationGross Motor Milestones in Idiopathic Clubfoot treated by Ponseti Method. Dr. Sameer Desai Pediatric Orthopedic Surgeon Pune
Gross Motor Milestones in Idiopathic Clubfoot treated by Ponseti Method Dr. Sameer Desai Pediatric Orthopedic Surgeon Pune Question asked by parent Will my baby walk When will my baby walk Aims and Objectives
More informationGM3 Synthase Deficiency A Potentially Treatable Disease?
GM3 Synthase Deficiency A Potentially Treatable Disease? DDC Clinic mission: To enhance the quality of life for people with special needs caused by rare genetic disorders DDC Clinic Started with a Family
More informationAdverse Outcomes After Hospitalization and Delirium in Persons With Alzheimer Disease
Adverse Outcomes After Hospitalization and Delirium in Persons With Alzheimer Disease J. Sukanya 05.Jul.2012 Outline Background Methods Results Discussion Appraisal Background Common outcomes in hospitalized
More informationAdvances in Alzheimer s diagnosis; implications for clinical practice. Wiesje M. van der Flier Alzheimer Europe Conference Berlin, 4 October 2017
Advances in Alzheimer s diagnosis; implications for clinical practice Wiesje M. van der Flier Alzheimer Europe Conference Berlin, 4 October 2017 Grand Challenge Alzheimer s disease 47 million worldwide
More informationNew NCCIH Funding Opportunities for Natural Product Clinical Trials. May 9, 2017
New NCCIH Funding Opportunities for Natural Product Clinical Trials May 9, 2017 The Webinar will start at 2:00 p.m. All participants have been placed on mute. We will take questions at the end of the presentation
More informationFramework for Defining Evidentiary Standards for Biomarker Qualification: Minimal Residual Disease (MRD) in Multiple Myeloma (MM)
Framework for Defining Evidentiary Standards for Biomarker Qualification: Minimal Residual Disease (MRD) in Multiple Myeloma (MM) MRD in Multiple Myeloma Team Biomarker Qualification Workshop Framework
More informationNeurofibromatosis Research Program
Neurofibromatosis Research Program Strategic Plan INTRODUCTION The Congressionally Directed Medical Research Programs (CDMRP) represents a unique partnership among the U.S. Congress, the military, and
More informationSpinal Muscular Atrophy as a Focus Indication for Biomarker Development. Meg Winberg, PhD Spinal Muscular Atrophy Foundation February 26, 2007
Spinal Muscular Atrophy as a Focus Indication for Biomarker Development Meg Winberg, PhD Spinal Muscular Atrophy Foundation February 26, 2007 Why SMA? p Low incidence, but a large orphan indication p Scientifically
More informationA Case Refort of Sandhoff Disease
Korean J Ophthalmol Vol. 19:68-72, 2005 A Case Refort of Sandhoff Disease Yie-Min Yun, MD, Su-Na Lee, MD Department of Ophthalmology, College of Medicine, Chungnam National University, Daejeon, Korea Sandhoff
More informationThe CCPH Featured Member is Cecil Doggette. Cecil is the Director of Outreach Services at Health Services for Children With Special Needs, Inc.
The CCPH Featured Member is Cecil Doggette. Cecil is the Director of Outreach Services at Health Services for Children With Special Needs, Inc. In his interview, Cecil describes the uniqueness of this
More informationTool to Assess Risk of Bias in Cohort Studies
Tool to Assess Risk of Bias in Cohort Studies Contributed by the CLARITY Group at McMaster University 1. Was selection of exposed and non-exposed cohorts drawn from the same population? Exposed and unexposed
More informationExampro A-level Biology
Exampro A-level Biology YEAR 12 BIOLOGY CHRISTMAS WORK Name: Class: Author: Date: Time: 54 Marks: 49 Comments: Page 1 of 12 Q1. (a) Describe how phospholipids are arranged in a plasma membrane.........
More informationNatural History of JNCL and other NCLs
Natural History of JNCL and other NCLs Jonathan W. Mink, MD PhD Departments of Neurology, Neurobiology & Anatomy, Brain & Cognitive Sciences, and Pediatrics University of Rochester Neuronal Ceroid Lipofuscinosis
More informationFinal Report. Supported Decision-Making: North Carolina.
Final Report Grant: National Resource Center for Supported Decision-Making s State Grant Program : North Carolina Project Period: 7/1/2015 6/30/2016 Date of Report: 7/29/2016 Project Title: Grantee Name:
More informationWell-Care for Children in Placement
Chapter 3 Well-Care for Children in Placement Introduction All children in placement are entitled to adequate well-child care and timely immunizations. The Early and Periodic Screening, Diagnosis, and
More informationEuropean Prevention of Alzheimer s Dementia (EPAD)
European Prevention of Alzheimer s Dementia (EPAD) Ron Marcus, MD ISCTM Adaptive Design Workshop February 20, 2018 1 EPAD Goal The European Prevention of Alzheimer's Dementia (EPAD) project aims to develop
More informationMRI-Based Biomarkers of Therapeutic Response in Triple-Negative Breast Cancer
MRI-Based Biomarkers of Therapeutic Response in Triple-Negative Breast Cancer Daniel Golden Postdoctoral Scholar (Radiology) Stanford University Daniel Rubin Laboratory NCI Cancer Imaging Fellowship Seminar
More informationProtocol Development: The Guiding Light of Any Clinical Study
Protocol Development: The Guiding Light of Any Clinical Study Susan G. Fisher, Ph.D. Chair, Department of Clinical Sciences 1 Introduction Importance/ relevance/ gaps in knowledge Specific purpose of the
More informationHematopoietic Stem Cell Transplantation for Adrenoleukodystrophy
Hematopoietic Stem Cell Transplantation for Adrenoleukodystrophy 2011 NHLBI Pediatric Workshop Sept. 14, 2011 Paul Orchard, M.D. Division of Pediatric Blood and Marrow Transplant University of Minnesota
More informationUNIVERSITY OF WASHINGTON
UNIVERSITY OF WASHINGTON THE FETAL ALCOHOL SYNDROME DIAGNOSTIC AND PREVENTION NETWORK (FAS DPN) Center for Human Development and Disability Dear Sir or Madam, Thank you very much for your request for an
More informationFatty Acids Synthesis L3
Fatty Acids Synthesis L3 The pathway for fatty acid synthesis occurs in the cytoplasm, whereas, oxidation occurs in the mitochondria. The other major difference is the use of nucleotide co-factors. Oxidation
More informationMinority populations are hardest hit by diabetes. Join us to Stop Diabetes in your community!
Minority populations are hardest hit by diabetes. Join us to Stop Diabetes in your community! Print Your Name Zip Write your email to receive Action Alerts! Are you a or? I want to support my community!
More informationMeasures. David Black, Ph.D. Pediatric and Developmental. Introduction to the Principles and Practice of Clinical Research
Introduction to the Principles and Practice of Clinical Research Measures David Black, Ph.D. Pediatric and Developmental Neuroscience, NIMH With thanks to Audrey Thurm Daniel Pine With thanks to Audrey
More informationSpinal Muscular Atrophy in 2017
Spinal Muscular Atrophy in 2017 Leigh Maria Ramos-Platt, MD Children s Hospital of Los Angeles University of Southern California Keck School of Medicine Disclosures MDA Care Center Grant In this presentation,
More informationSection 2: Multivariate or Multiple Domain Methods Aldurazyme Responder Index
Disclaimer: Presentation slides from the Rare Disease Workshop Series are posted by the Kakkis EveryLife Foundation, for educational purposes only. They are for use by drug development professionals and
More informationEmil D. Kakkis, M.D., Ph.D. President Kakkis EveryLife Foundation
Disclaimer: Presentation slides from the Rare Disease Workshop Series are posted by the EveryLife Foundation for Rare Diseases for educational purposes only. They are for use by drug development professionals
More informationPrincipal Theories in Adapted Physical Activity. Claudine Sherrill, CAPE
Principal Theories in Adapted Physical Activity Claudine Sherrill, CAPE Dr. Eliane Mauerberg-deCastro, 2005 Greetings from The International Federation of Adapted Physical Activity (IFAPA) Founded in 1973
More informationDISCLOSURES. Objectives. THE EPIDEMIC of 21 st Century. Clinical Assessment of Cognition: New & Emerging Tools for Diagnosing Dementia NONE TO REPORT
Clinical Assessment of Cognition: New & Emerging Tools for Diagnosing Dementia DISCLOSURES NONE TO REPORT Freddi Segal Gidan, PA, PhD USC Keck School of Medicine Rancho/USC California Alzheimers Disease
More informationBarriers to Successful Implementation of Priority Population Programming: Lessons Learned from the Health Smiles Program in Brant County
of Priority Population Programming: Lessons Learned from the Health Smiles Program in Brant County Sinton, J. & Vamos, C. Background Ontario s Poverty Reduction Strategy Background Financing of dental
More informationIncorporating Clinical Information into the Label
ECC Population Health Group LLC expertise-driven consulting in global maternal-child health & pharmacoepidemiology Incorporating Clinical Information into the Label Labels without Categories: A Workshop
More informationFASD - Frontline EFAN. Inside this issue: 4 Fun Ways to Spend Mother s Day. Go to the movies: Get Pampered: Make Lasting Impressions
FASD - Frontline EFAN May 2015 Inside this issue: FASD Success Story Your Brain on Mediation The Paper Trail Notebook 2 2 3 4 Fun Ways to Spend Mother s Day How do you spend your special day, Mom? Get
More informationBOLD Based MRI Functional Connectivity December 2, 2011
BOLD Based MRI Functional Connectivity December 2, 2011 Luigi Maccotta, MD, PhD Adult Epilepsy Center Washington University School of Medicine American Epilepsy Society Annual Meeting Support Disclosure
More informationNon Alcoholic Fatty Liver Disease (NAFLD) - Global API Manufacturers, Marketed and Phase III Drugs Landscape, 2018
Non Alcoholic Fatty Liver Disease (NAFLD) - Global API Manufacturers, Marketed and Phase III Drugs Landscape, 2018 Non Alcoholic Fatty Liver Disease (NAFLD) - Global API Manufacturers, Marketed and Phase
More informationHealth System Preparedness of Six EU Countries for a Future Alzheimer s Treatment. European Health Forum Gastein Soeren Mattke Oct 3, 2018
Health System Preparedness of Six EU Countries for a Future Alzheimer s Treatment European Health Forum Gastein Soeren Mattke Oct 3, 2018 Why focus on Alzheimer s treatment now? After many failures, guarded
More informationSibling studies reveal early signs of autism
NEWS Sibling studies reveal early signs of autism BY VIRGINIA HUGHES 4 APRIL 2008 1 / 6 Baby talk: By the end of their first year, infants who go on to develop autism already show distinct features of
More informationAmyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry
Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry DRAFT GUIDANCE This guidance document is being distributed for comment purposes only. Comments and suggestions regarding
More informationMaternal Influenza Immunization at WHO
Maternal Influenza Immunization at WHO Dr. Justin R. Ortiz Medical Officer Initiative for Vaccine Research GAP Partners Meeting Dubai, UAE 26 March 2014 WHO Position Paper Mother s Gift Trial GAVI Review
More informationNigeria: WHO and UNICEF estimates of immunization coverage: 2017 revision
Nigeria: WHO and UNICEF estimates of immunization coverage: 2017 revision July 7, 2018; page 1 WHO and UNICEF estimates of national immunization coverage - next revision available July 15, 2019 data received
More informationReadings: Required book: Batinovich, Sara Laufer. Sound Sense: Living and learning with hearing loss Gallaudet University Press, Washington D.C.
Office: SLHS 523 Tel: 626-0111 Email: jdean1@email.arizona.edu Office hours: TBA SLHS 255: Hearing, Health and Society James Dean, AuD, MED Dept. Speech, Language & Hearing Sciences School of Mind, Brain
More informationProposal form for the evaluation of a genetic test for NHS Service Gene Dossier
Proposal form for the evaluation of a genetic test for NHS Service Gene Dossier Test Disease Population Triad Disease name and description (please provide any alternative names you wish listed) (A)-Testing
More informationLOGIC MODEL TEMPLATE ACT EARLY SUMMIT -AK
Participant affiliations CAAI Title V Alaska Native Tribal Health Consortium EPSDT Coordination Governor s Council Part C UCEDD Stone Soup Group Special Education Service Agency Alaska Autism Resource
More informationCommunity Benefit Strategic Implementation Plan. Better together.
Community Benefit Strategic Implementation Plan 2016 2019 Better together. Table of Contents Introduction... 4 Priority 1: Community Health Infrastructure... 5 Objective 1.1: Focus resources strategically
More informationAlaska Division of Public Health Section of Women s, Children s and Family Health MCH Epidemiology Unit
Sandy Collins, MPH, RD Alaska Coordinator Janine Schoellhorn, MS, MPH Lead Epidemiologist and Data Analyst Alaska Division of Public Health Section of Women s, Children s and Family Health MCH Epidemiology
More informationALZHEIMER'S DISEASE PREVENTION TRIALS
ALZHEIMER'S DISEASE PREVENTION TRIALS Laurie Ryan, PhD Chief, Dementias of Aging Branch & Program Director, Alzheimer s Disease Clinical Trials Division of Neuroscience National Institute on Aging/National
More informationCongenital Heart Disease in the United States of America: Guidelines
Congenital Heart Disease in the United States of America: American Academy of Pediatrics Guidelines Christopher S. Snyder, MD, FAAP Chair of Section of Cardiology and Cardiovascular Surgery (AAP) Chief
More informationWe seek to impact individuals with Autism Spectrum Disorders in future by serving families and professionals in Central Kentucky.
Advocates for Autism Awareness Sample Advocates for Autism Awareness is a group of families providing hope and support to one another; striving to build partnerships between families & professionals; and
More informationQuality Improvement Approaches to BPD. Jay P. Goldsmith, M.D. Tulane University New Orleans, Louisiana
Quality Improvement Approaches to BPD Jay P. Goldsmith, M.D. Tulane University New Orleans, Louisiana goldsmith.jay@gmail.com No conflicts of interest to declare There is nothing more dangerous to the
More informationBackground: AAP and the UN Foundation Campaign Global Vaccine Advocacy Education Materials Program
Background: AAP and the UN Foundation Shot@Life Campaign Global Vaccine Advocacy Education Materials Program WHAT IS THE GOAL OF THE AAP AND SHOT@LIFE PROGRAM? The goal of the program is to raise awareness
More informationInsults to the Developing Brain & Effect on Neurodevelopmental Outcomes
Insults to the Developing Brain & Effect on Neurodevelopmental Outcomes Ira Adams-Chapman, MD Assistant Professor of Pediatrics Director, Developmental Progress Clinic Emory University School of Medicine
More informationNational Tay-Sachs & Allied Diseases Association Keeping Up with NTSAD, April 2013
National Tay-Sachs & Allied Diseases Association Keeping Up with NTSAD, April 2013 In This Issue In Others' Words Award Honorees Research Update Living with LOTS Day of Hope In the News Living with Grief
More informationProject 3dii: Expansion of the Home Environmental Asthma Management Program
1 Project 3dii: Expansion of the Home Environmental Asthma Management Program Asthma Primary Care Project Participation Opportunity Purpose 2 This Project Participation Opportunity is specifically targeted
More information5. Hospitals will provide the family with a copy of the Michigans Community Program: Information for Parents (MDCH /01). Copies can be ordered,
GUIDELINES FOR HEARING SCREENING A goal of newborn hearing screening is to ensure that all newborns receive a hearing screen before discharge from the hospital or before one month of age. All hospitals
More informationReturning HIV-exposed infants to care in Lilongwe, Malawi
Returning HIV-exposed infants to care in Lilongwe, Malawi Results from a pilot integrating infant defaulter tracing into the Malawi Option B+ programme Esmie Kamanga, Grace Banda, Innocent Mofolo, Gertrude
More informationEpidemiology: Overview of Key Concepts and Study Design. Polly Marchbanks
Epidemiology: Overview of Key Concepts and Study Design Polly Marchbanks Lecture Outline (1) Key epidemiologic concepts - Definition - What epi is not - What epi is - Process of epi research Lecture Outline
More informationSAFEMINDS EVALUATION OF IACC BUDGET Green denotes SafeMinds Recommendations, Red denotes IACC future action. IACC Budget Recommendation 5,300,000
Green denotes SafeMinds s, Red denotes IACC future action 1. When Should I Be Concerned? ST 1.1 Develop, with existing tools, at least one efficient diagnostic instrument (e.g., briefer, less time intensive)
More informationState of the State. Massachusetts 2010
State of the State Massachusetts 2010 Child Count Massachusetts AGES Children with Autism in Massachusetts 1999-2000 Children with Autism in Massachusetts 2007-2008 3-5 yrs 231 1,839 6-11 yrs 290 3,796
More informationLearn the steps to identify pediatric muscle weakness and signs of neuromuscular disease.
Learn the steps to identify pediatric muscle weakness and signs of neuromuscular disease. Listen Observe Evaluate Test Refer Guide for primary care providers includes: Surveillance Aid: Assessing Weakness
More informationNCCIH Natural Products Technical Assistance Webinar: Exploratory Clinical Trials and Studies of Natural Products
NCCIH Natural Products Technical Assistance Webinar: Exploratory Clinical Trials and Studies of Natural Products October 26, 2016 Moderator: Catherine Law Presenters: Ashlee Tipton, PhD; Wendy Weber, ND,
More informationAccelerating Translation at Dana-Farber Cancer Institute*
Accelerating Translation at Dana-Farber Cancer Institute* *and our partner institutions February 12, 2013 Edward J Benz JR, MD Dana Farber Cancer Institute 1 2 Academic Structure All Dana-Farber faculty
More informationDisclosures. Learning Objectives. Mechanical Ventilation of Infants with Severe BPD: An Interdisciplinary Approach 3/10/2017
Mechanical Ventilation of Infants with Severe BPD: An Interdisciplinary Approach Steven H. Abman, MD Professor, Department of Pediatrics Director, Pediatric Heart Lung Center University of Colorado School
More informationRepeat Pregnancies and HIV Care Engagement among Postpartum HIV-infected Women in Atlanta, Georgia,
Repeat Pregnancies and HIV Care Engagement among Postpartum HIV-infected Women in Atlanta, Georgia, 2011-2015 Anandi N. Sheth, Christina M. Meade, Martina Badell, Susan A. Davis, Stephanie Hackett, Joy
More informationChallenges of Observational and Retrospective Studies
Challenges of Observational and Retrospective Studies Kyoungmi Kim, Ph.D. March 8, 2017 This seminar is jointly supported by the following NIH-funded centers: Background There are several methods in which
More informationMake memories matter at the Investors Group Walk for Alzheimer s!
April June 2018 See inside for dementia programs in the Central Interior. Make memories matter at the Investors Group Walk for Alzheimer s! Join us at the Investors Group Walk for Alzheimer s on Sunday,
More informationInformation Processing Mid-February 2007
Information Processing Mid-February 2007 Information Processing The Information Processing Approach Speed of Processing Attention What Is the Information-Processing Approach? Focuses on ways people process
More informationStandardized description of rare diseases the natural course and treatment of metachromatic leukodystrophy
Standardized description of rare diseases the natural course and treatment of metachromatic leukodystrophy Samuel Groeschel, Christiane Kehrer, Ingeborg Krägeloh-Mann Department of Paediatric Neurology
More informationLeveraging LTSAE/SPAN Partnership for Collective Impact in New Jersey
Leveraging LTSAE/SPAN Partnership for Collective Impact in New Jersey Diana Autin - Executive Co-Director, SPAN Deepa Srinivasavaradan - CDC s Act Early Ambassador to NJ SPAN s Vision & Mission VISION
More informationGenetic Tests and Genetic Counseling How to Analyze Your Own Genome
Genetic Tests and Genetic Counseling 02-223 How to Analyze Your Own Genome Genetic Tests for Huntington Disease Hun7ngton Disease Incurable brain disorder that runs in families Movement, cogni7ve, and
More informationModeling of early-infant brain growth using longitudinal data from diffusion tensor imaging.
Modeling of early-infant brain growth using longitudinal data from diffusion tensor imaging. Guido Gerig, Neda Sadeghi, PhD, Marcel Prastawa, Tom Fletcher, Clement Vachet Scientific Computing and Imaging
More informationAfghanistan: WHO and UNICEF estimates of immunization coverage: 2017 revision
Afghanistan: WHO and UNICEF estimates of immunization coverage: 2017 revision July 7, 2018; page 1 WHO and UNICEF estimates of national immunization coverage - next revision available July 15, 2019 data
More informationToronto Declaration: Strategies to control and eliminate viral hepatitis globally. A call for coordinated action
Toronto Declaration: Strategies to control and eliminate viral hepatitis globally A call for coordinated action Hepatitis B National Action Plan All countries should develop a national and/or regional
More informationIdentification & Diagnosis of Cryptogenic Stroke What is NINDS/role
Identification & Diagnosis of Cryptogenic Stroke What is NINDS/role October 9, 2015 Scott Janis, Ph.D. Program Director, National Institute of Neurological Disorders and Stroke, The National Institute
More information