MLD Family Conference™ ... Franklin, TN - 2019

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2019 MLD Family Conference™ AGENDA

Sponsored by MLD Foundation

June 28th–29th in Franklin, Tennessee

as of June 23, 2019 – subject to change ... See last year's agenda here


Friday, June 28th


Friday, June 28th

9:00am

Registration opens
Respite Room opens
Light Continental Breakfast

10:00

Welcome

Dean Suhr, President, MLD Foundation **
Teryn Suhr, Executive Director, MLD Foundation **

10:15

What’s this Thing Called MLD? – An overview of MLD, it’s biology, diagnostics, and therapies ... lysosomal disease, natural history, genotype/phenotype correlations, biomarkers

facilitated by
Brett Crawford Ph.D., BioMarinmember of one of the MLD Boards
Marc Patterson MD, Mayo Clinic member of one of the MLD Boards **
Dean Suhr –  MLD Foundation

12:00

Lunch (Friday Industry and academic experts photo)

 

1:00

Research & Clinical Trials – learn the steps, hear and
discuss the latest in MLD research
- Natural History Studies
- MLD Patient Powered Registry
- Clinical Trials ... Enzyme Replacement, Gene Therapy, & Transplant
- Other BioTech/Pharma ... increasing & ongoing interest

Discussion ... how you can participate in better understanding MLD, help & next steps

  • • Clinical Trials
  •    - Cell Therapy
  •    - Gene Therapy
  •    - Enzyme Replacement Therapy (ERT)
  •    - Substrate Reduction Therapy
    - Other BioTech/Pharma ... increasing & ongoing interest

Discussion ... how you can help & next steps

continued...

facilitated by
Brett Crawford Ph.D.
, BioMarinmember of one of the MLD Boards
Marc Patterson MD, Mayo Clinicmember of one of the MLD Boards **

Laura Adang MD PhD, CHOP – Children's Hospital of Philadelphia
Francesca Fumagalli MD, San Raffaele/TIGET (Italy) - via video
Steve Glyman MD, Homology Medicines
Leslie Jacobsen MD, Takeda Therapeutics
Paola Luzi, Jefferson University Lysosomal Lab
Yuriy Shapovolv,
Orchard Therapeutics

Discussion - All

3:00

Break

3:15

Ladies Breakout, Men’s Breakouts – A time for those new and for those more experienced on the MLD journey to come together in a small, caring, confidential environment

Academia & Industry Breakout– Research presentation – Discovery & Characterization of Novel CGT Inhibitors – SRT for MLD & Krabbe; brainstorm of priorities for future MLD research, clinical, and advocacy efforts.

MLD Parents and Caregivers


Academia, Institutional, & Industry Guests
Please note: The research presentation is not authorized for patient/parent attendance, a paper will be published.

5:00

Family Time

 

6:30

Dinner

 

9:00-12:00

MLD After Hours ...  ... You are invited to “MLD After Hours”. Kick off your shoes and join us for some informal conversation and sharing.

 


Saturday, July 29th

8:00am – 9:30

Buffet Breakfast

 

8:45

Registration opens
Respite Room opens

 

9:00

MLD Newborn Screening Academy

  • • Status of MLD screen validation pilot studies
  • • Federal RUSP application process & status
  • • What to expect at the state level
  • • and most importantly how we can and must all work with a unified passion to get MLD NBS launched in each state and country. It's time for families to effectively engage!

Dean Suhr, MLD Foundation **
Teryn Suhr, MLD Foundation **

Jessica Daiker, Research Scientist Gelb lab – Univ. of Washington
Joan Keutzer PhD, formerly of Sanofi Genzyme via videomember of one of the MLD Boards
Paola Luzi, Jefferson University Lysosomal Lab
Melissa Wasserstein MD, Montifiore, Einstein College of Medicine via video

Discussion - All

10:45

Break

 

11:00

Group Photo

 

11:15

Remembering Our MLD Angels – Butterfly Ceremony – A special remembrance of those who have earned their Butterfly Wings since our last time together ... and honoring all of our MLD Angels

All attendees ... we will live stream the ceremony

12:00

Lunch

 





1:00

MLD Family Care ... the entire afternoon is devoted to helping you improve the quality of life for your MLD loved one, you, and your family. The agenda will be fluid as we adapt to your personal questions and needs.

Meeting the Needs of Siblings – from a developmental perspective






Jenni Rogers, RN
– Phoenix Children’s via video

2:00

What’s Up Doc? Improving communications between patients, families, and healthcare professionals

Dawn A Laney, MS CGC CCRC - Emory/ThinkGenetics

2:45

break

 

3:00

Complex Care Plans – What they are & how to use them

Dawn A Laney, MS CGC CCRC - Emory/ThinkGenetics

3:45

What’s On Your Mind? Open discussion about issues on attendee’s mind. Topics like “storming” ... what it is, causes, treatments ...

facilitated by Marc Patterson MD, Mayo Clinic
Everyone

4:30

Group Discussion & Wrap Up

Everyone

5:15

Family time

 

6:45

Parade of Champions ... call time

 

7:00

Parade of Champions Grand Entrance and Dinner

 

10:00-12:00

MLD After Hours ... Please join us for “MLD After Hours”. Kick off your shoes and join us for some informal conversation and sharing.

 


tbd - to be determined

member of one of the MLD Boards Member of MLD Foundation's Medical and Scientific Advisory Board (MSAB) or the ** MLD Foundation Board (fiduciary)



Sunday, June 30th

Sunday is an optional social day.
Hotel checkout is at 12pm
.

Thank you to our sponsors, service, & grant providers:

Honoring Olivia   Takeda   Orchard Therapeutics   Homology Medicines   Believing For Bryleigh  
who through their sponsorships, grants, and support are making this conference much more affordable for the attending families.

We also wish to thank the individual families for their donations to the scholarship fund.


Industry and Academia Attendees

Name Institution Location Title/Specialty

Laura Adang MD PhD

Children’s Hospital Philadelphia

Philadelphia, PA

Neurologist, Clinical Researcher

Randell Carmer

Soulumination

Seattle, WA

Photographer

Brett Crawford PhDmember of one of the MLD Boards

BioMarin

Novato, CA

Staff Scientist

Jessica Daiker

Gelb Lab

Seattle, WA

Research Scientist

Francesca Fumagalli, MD

San Raffaele Hospital, Telethon Institute for Gene Therapy (SR-TIGET)

Milano, Italy

Neurologist Consultant (via video)

Steve Glyman MD

Homology Medicines

Bedford, MA

Vice President, Clinical Development

Christine Hoffmann **

MLD Family Champions
Co-Coordinator & MLD Family member

Scottsdale, AZ

Vice President, Clinical Development

Leslie Jacobsen, MD

Takeda Therapeutics

Lexington, MA

Global Clinical Development Lead Rare Metabolic Diseases

Dawn Laney, MS CGC CCRC

Emory Genetics / ThinkGenetics

Atlanta, GA

Genetic Counselor

Paola Luzi PhD

Jefferson University Lysosomal Lab

Philadelphia, PA

Lab Associate Director, Asst. Professor

Joan Keutzer PhD *

retired, formerly of Sanofi Genzyme

Cambridge, MA

formerly Vice President and Head, Global Strategic Services, Rare Diseases

Tara Mathiesen, RN BSN

Orchard Therapeutics

Bedford, MA

SVP, Corporate Communications & Patient Advocacy

Bruce Oliver, PhD

Orchard Therapeutics

Rochester, MN

Sr. Director, Clinical Scientist

Marc Patterson MDmember of one of the MLD Boards **

Mayo Clinic

Boston, MA

Professor of Neurology

Jenni Rogers, RN

Children’s Hospital Phoenix

Phoenix, AZ

Registered Nurse

Courtney Shook

MLD Family Respite Room Coordinator & MLD Family member

Springfield, PA

Aunt to Kendall Williamson

Yuriy Shapovalov MD

Orchard Therapeutics

Boston, MA

Head, Global Medical Affairs, Neurometabolic Conditions

Dean Suhr

MLD Foundation
Dad to Lindy and angel Darcee

West Linn, OR

President & co-Founder

Teryn Suhr

MLD Foundation
Mom to Lindy and angel Darcee

West Linn, OR

Executive Director & co-Founder

Melissa Wasserstein MD

Children’s Hospital Montefiore

Bronx, NY

Chief, Division of Pediatric Genetics

member of one of the MLD Boards Member of MLD Foundation's Medical and Scientific Advisory Board (MSAB) or the ** MLD Foundation Board (fiduciary)

Questions?
Contact Dean Suhr <deansuhr@MLDfoundation.org> +1 503-656-4808, USA: 1-800-617-8387, or FAX +1 503-212-0159

This conference is organized by MLD Foundation. Check with your tax advisor about the deductibility of any expenses associated with this conference.