Stem Cell Transplantation:A Donor for Every Patient1. Discuss haploidentical donor stem cell transplantation (SCT).2. Explain the advantages and disadvantages of haploidentical donor SCT.3. Discuss recent outcomes with CD34-selected alternative donor SCT.

Progress in Transplant for Sickle Cell Disease: The Nursing Perspective1. List indications for hematopoietic progenitor cell transplantation (HPCT) for individuals with sickle cell disease (SCD).2. Discuss unique principles of HPCT for SCD.3. Describe current and emerging trends in HPCT for SCD.

Applications of Family-Centered Care in Hem-Onc and BMT Setting1. Examine the core principles of Patient- and Family-Centered Care.2. Define the practice of Family-Centered Rounds.3. Recognize the applications of PFCC with oncology and BMT families.

The Moral Imperative to Implement New Therapies for Childhood Cancer: How T Cells Can Be Infused as a Drug1. Develop novel and combination immunotherapy strategies for patients resistant to available targeted therapies in practice. 2. Integrate resources available using non-viral gene transfer programs, in the design and conduct of novel clinical trials. 3. Evaluate quality metrics for T cell therapy to improve patient outcomes.

Racial/Ethnic Differences in Outcome of Children with Acute Lymphoblastic Leukemia:A 360-Degree Approach1. Describe disparities in outcome in child with ALL.2. Recognize the underlying causes of disparities.3. Describe targeted interventions to mitigate disparities.

Developing Best Practice Recommendations for Patient/Family Education in Pediatric Oncology: The Children’s Oncology Group Experience1. Define key knowledge gaps regarding patient/ family education need in newly diagnosed pediatric oncology patients.2. Describe current evidence to inform best practices for patient/family education in pediatric oncology.3. Explain the process used for developing best practice recommendations for patient/family education within the Children’s Oncology Group.

Panel DiscussionIs More Better? A Look Back on Nearly Two Decades of Using High Dose Chemotherapy to Treat Pediatric Brain Tumors1. Describe traditional treatment and outcome for young children with malignant brain tumors.2. Explain the theoretical role of high-dose chemotherapy as a means of avoiding radiation and improving survival.3. Review institutional experience and recent clinical trials that treat young children with malignant brain tumors.4. Describe challenges for family and healthcare providers caring for children with highly malignant brain tumors.

This conference is made possible by a generous gift from the Tucker Beam family.

Upon completion of this live activity, participants will be able to:

Continuing EducationChildren’s of Alabama designates this live activity for a maximum of 5.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Children’s of Alabama is accredited by the Medical Association of the State of Alabama to provide continuing medical education for physicians.

BEYOND THE DIAGNOSIS ART EXHIBITSelected works from this traveling art exhibit, focusing on the rare disease patient, will be displayed at Children’s of Alabama from February 20 through March 17, 2017. Art has been used for thousands of years to successfully convey a message, whether it be a story or a glimpse into the human spirit. The purpose of this exhibit, presented by the Rare Disease United Foundation, is to encourage a look “beyond the diagnosis” to the patient. Two works from the exhibit are shown in this brochure, including the one above —"Ashlyn" (STURGE-WEBER SYNDROME) by Wolfgang Widmoser .

About Our Keynote SpeakerDebbie Krakow, MD

Professor, Orthopaedic Surgery, Human Genetics and Obstetrics and Gynecology

David Geffen School of Medicine, UCLALos Angeles, CA

Dr. Krakow is an internationally recognized expert on the diagnosis and management of skeletal disorders. She is Professor of Human Genetics and Orthopaedic Surgery at UCLA. She also is Co-Director of the UCLA International Skeletal Dysplasia Registry, a referral center for that focuses on clinical diagnoses and the molecular biology of genetic bone diseases. In addition to her clinical work as a medical geneticist and maternal fetal medicine physician, Dr. Krakow directs a laboratory that has identified the genetic and cellular basis of skeletal diseases. She has authored over 100 peer reviewed papers, numerous reviews and book chapters.

8:00-8:30 Registration and Continental Breakfast Provided

8:30-8:35 Welcome Bruce R. Korf, MD, PhD

SESSION 1 INDICATIONS FOR REFERRAL8:35-10:00 Nathanial Robin, MD - Moderator

8:35-9:10 Big Size, Small Size: Genetic Causes of Growth Differences Nathaniel Robin, MD University of Alabama at Birmingham 9:10-9:45 Physical Clues to Explain Developmental Differences Chad Haldeman-Englert, MD Mission Health, Asheville, NC 9:45-10:00 Questions and Panel Discussion NHR, CH-E, ACHE

10:00-10:15 Break

SESSION 2 DIAGNOSTIC APPROACHES10:15-11:45 Anna C.E. Hurst, MD, MS - Moderator

10:15-10:45 Cytogenetic Testing in the Genetic Evaluation Fady Mikhail, MD, PhD University of Alabama at Birmingham

10:45-11:25 Molecular Genomic Testing Strategies David Bick, MD HudsonAlpha, Huntsville, AL

11:25-11:45 Practical Resources for the Pediatrician Anna C.E. Hurst, MD, MS University of Alabama t Birmingham

Agenda Friday, March 3, 2017

11:45-12:45 KEYNOTE ADDRESS Genotype Does Not Always Explain Phenotype for Skeletal Disorders Debbie Krakow, MD David Geffen School of Medicine, UCLA

12:45-1:00 Break

LUNCH/ SESSION 3 PARENT PANEL1:00-2:00 Ashley Cannon, PhD, MS, CGC - Moderator University of Alabama at Birmingham SESSION 4 OUTCOMES AND FOLLOW-UP2:00-3:30 Bruce R. Korf, MD, PhD - Moderator

2:00-2:30 Update on the UAB Undiagnosed Diseases Program Bruce R. Korf, MD, PhD University of Alabama at Birmingham

2:30-3:00 Outcomes of Genetic Counseling Following Genome Sequencing: Experience From the HudsonAlpha Clinical Sequencing Exploratory Research project Whitley Kelley, MS HudsonAlpha, Huntsville, AL

3:00-3:30 Ethical and Legal Issues in Diagnostic Genome Sequencing Tom May, PhD HudsonAlpha, Huntsville, AL

Learning ObjectivesUPON COMPLETION OF THIS LIVE ACTIVITY,

PARTICIPANTS WILL BE ABLE TO:

• Recognize features of a child who may benefit from a genetic evaluation.

• Identify the benefits and limitations of chromosomal microarray array testing.

• Distinguish when a child may benefit from broad molecular-based testing strategies (such as whole exome or whole genome sequencing).

• Utilize practical resources for the healthcare management of a child with a genetic condition.

• Reflect on the psychosocial needs of the family of a child with a genetic condition.

• Review outcomes from existing genetic programs in the region.

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GENETICS

Chi ldrensAL.org/genet ics

March 3, 2017Friday, 8:00am — 3:30pm

Bradley Lecture Center4th Floor, Children's Harbor Building

1600 6th Avenue SouthBirmingham, AL 35233

SpeakersFOR ONLINE REGISTRATION, please visit

ChildrensAL.org/genetics

Or complete the form below and mail, email or fax registration form and payment to:

Shaila Handattu, PhD, MBADepartment of Genetics

Kaul 230, 720 20th Street SouthBirmingham, AL 35294

hande@uab.edu FAX 205.934.9488

$35 to AttendPlease make checks payable to Children’s of Alabama.

NAME_______________________________________________________

A D D R E S S ___________________________________________

P H O N E _____________________________________________

F A X _________________________________________________

E M A I L _______________________________________________

_____ PHYSICIAN _____ NURSE

_____ Check here if a Children’s or UAB employee.

Symposium is free to Children's of Alabama

and UAB employees, but you must show your

ID badge to enter.

Special dietary needs__________________________________________

_______________________________________________________

Parking will be availablein the Children’s Park Place Deck.

The entrance to the deck is at 16th Street South and 5th Avenue South.

Questions? Contact Shaila Handattu at hande@uab.edu

Registration

GENETICS AND GENOMICS IN DAY TO DAY

MEDICAL PRACTICE FOURTH ANNUAL RARE DISEASE GENOMICS SYMPOSIUM

David Bick, MDMedical Director, HudsonAlpha Medical Genomics ProgramHuntsville, AL

Ashley Cannon, PhD, MS, CGCInstructor, Certified Genetic CounselorDepartment of GeneticsUniversity of Alabama at Birmingham (UAB)

Chad Haldeman-Englert, MDClinical Geneticist, Fullerton Genetics CenterMission Health, Asheville, NC

Anna Chesson Edens Hurst, MD, MSAssistant Professor, Department of GeneticsUniversity of Alabama at Birmingham (UAB)

Whitley V. Kelley, MSGenetic Counselor, Clinical Applications SpecialistHudsonAlpha Institute for BiotechnologyHuntsville, AL

Bruce R. Korf, MD, PhDProfessor and Chair, Department of GeneticsDirector, Heflin Center for Genomic SciencesCo-Director, UAB-HudsonAlpha Center for Genomic MedicineUniversity of Alabama at Birmingham (UAB)

Deborah Krakow, MDProfessor, Orthopaedic Surgery, Human Genetics and Obstetrics and GynecologyDavid Geffen School of Medicine, UCLALos Angeles, CA

Thomas May, PhDSenior ScientistHudsonAlpha Institute for BiotechnologyHuntsville, AL

Fady M. Mikhail, MD, PhDAssociate Professor and Co-DirectorClinical Cytogenetics Laboratory, Department of GeneticsUniversity of Alabama at Birmingham (UAB)

Nathaniel H. Robin, MDProfessor of Genetics and PediatricsUniversity of Alabama at Birmingham (UAB)

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