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

See the Conference Overview



2019 MLD Family Conference™ AGENDA

Sponsored by MLD Foundation

June 28th–29th in Franklin, Tennessee

as of May 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 & Personal Introductions

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

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 * **
Discussion - All

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 & the MLD Patient Powered Registry Clinical Trials ... Enzyme Replacement, Gene Therapy, & Transplant Other BioTech/Pharma ... increasing interest
Discussion ... how you can participate in better understanding MLD, help & next steps

  • • Natural History Studies
  • • MLD Patient Powered Registry
  • • 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
Maria Escolar MD, NDRD, Children's Hospital Pittsburgh
Francesca Fumagalli MD, San Raffaele/TIGET (Italy) - via video
Steve Glyman MD, Homology Medicines
Leslie Jacobsen MD, Takeda Therapeutics
Yuriy Shapovolv, Orchard Therapeutics
Paul Szabolcs MD, Children's Hospital Pittsburgh

Discussion - All

2:30

Local Care Teams & Resources – How to benefit from a Center of Excellence. What to do when you can’t make it to a LCN or MLD specialists.


Panel
Laura Adang MD PhD
, CHOP – Children's Hospital of Philadelphia
Maria Escolar MD, NDRD, Children's Hospital Pittsburgh
tbd

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– A brief brainstorm of priorities for future MLD research, clinical, and advocacy efforts

MLD Parents and Caregivers


Academia and Industry Attendees

5:00

Family Time

 

6:00

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. Who knows, you might even find some surprises to pass the time!

 


Saturday, July 29th

8:00am – 9:30

Buffet Breakfast

 

9:00

Registration opens
Respite Room opens

 

9:30

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 **
Joan Keutzer PhD, retired (as of June 21st), formerly of Sanofi Genzyme (tentative)
Melissa Wasserstein MD, Montifiore, Einstein College of Medicine (tentative)
Jessica Daiker, Research Scientist Gelb lab – Univ. of Washington

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

 

1:15

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.

Discussions and Sessions will include:
-
Patient Empowerment
- Building a Complex Care Plan
- Common MLD care issues

-
Optimizing clinical care and care strategies
- Hands on PT session

panels & discussion
Dawn A Laney, MS CGC CCRC - Emory/ThinkGenetics
tbd
tbd

Everyone

2:45

break

 

3:00

MLD Family Care ... continued

all

4:30

Group Discussion & Wrap Up

Everyone

5:15

Family time

 

7:00

MLD Family kiddos Grand Entrance and Introductions

 

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. Who knows, you might even find some surprises to pass the time!

 


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  
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

Brett Crawford PhD *

BioMarin

Novato, CA

Staff Scientist

Maria Escolar MD *

Program for the Study of Neurodevelopment in Rare Disorders

Pittsburgh, PA

Developmental Pediatrician, Director - NDRD - Children’s Hospital Pittsburgh

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)

Christine Harrison

Orchard Therapeutics

Boston, MA

VP, Public Affairs and Stakeholder Engagement

Francesca Fumagalli MD

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

Milano, Italy

Neurologist Consultant

Steve Glyman MD

Homology Medicines

Bedford, MA

Vice President, Clinical Development

Leslie Jacobsen, MD

Takeda Therapeutics

Lexington, MA

Global Clinical Development Lead Rare Metabolic Diseases

Poala Luzi PhD

Jefferson University Lysosomal Lab

Philadelphia, PA

Lab Associate Director, Asst. Professor

Brad Mathis

Orchard Therapeutics

Bedford, MA

US GM & head of Commercial Operations

Theresa McNeely

Homology Medicines

Bedford, MA

SVP, Corporate Communications & Patient Advocacy

Marc Patterson MD * **

Mayo Clinic

Rochester, MN

Professor of Neurology

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

Paul Szabolcs MD

Univ. of Pittsburgh Medical Center

Pittsburgh, PA

Chief, Blood and Marrow Transplant & Cellular Therapy

Randell Walton

Soulumination

Seattle, WA

Photographer and storyteller

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.