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MLD Family Conference™ ... Parsippany, NJ - 2013

See the Conference Overview

Registration Closed
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  Online registration for this conference closed on Monday, July 15. If you would still like to register we will try to accommodate you as best we can. Please contact us via email or at +1 503-656-4808.

LIVE WEBCAST: There will be plenty of space on the free live webcast, which will appear on the Conference Overview page during the conference.

*** We are sorry, due to planning and space constraints we cannot accommodate on-site registration or walk-in attendees. ***

See the Conference Invitation


MLD Family Conference™ AGENDA

Sponsored by the MLD Foundation

Aug. 2-3rd in Parsippany, New Jersey

as of July 29, 2013 – subject to change ... See last year's agenda here

Friday, August 2nd

12:30

Doors open - sign in & registration,
respite room opens

Hilton Parsippany
click for map

1:00

Welcome & Introductions

Dean Suhr
President, MLD Foundation

MLD 101 ... an overview of Metachromatic Leukodystrophy


2:00


What is MLD?

- Biochemistry, Neurology, Genetics, Progressions

- Genotype/Phenotype Correlations ... what do the mutations tell us about the progression of MLD? What should we study?

- Tracking the progression ... why should we track the disease progression and what are best methods ... impact on research and practical care

- Genetics of MLD ... facts, figures, and awareness for the extended family


PANEL:

Dr. David Wenger - Director, Lysosomal Diseases Testing Laboratory, Philadelphia, PA

Dr. Lawrence Charnas
- Consultant Neurogenetics, Kennedy Krieger Institute, Baltimore, MD


Sally Ann Rodríguez
ScM - Associate Genetic Counselor, Recombine, Livingston NJ

Aliza (Ricklis) Zidell, MS – Genetic Counselor, Hackensack University Medical Center

3:00  break
3:15 A Primer ... Research, Trials, Therapies, Scientific Studies vs. Patient Reported Observations tbd


4:00


Newborn Screening for Lysosomal Diseases ...
it takes more than a screening test ...


Dean Suhr



SSI, Medicaid, etc.


Social Security (invited)



What Role Do You Want for the MLD Foundation



Dean Suhr


5:30


Dream Foundation – fulfilling wishes for adults


Kristy Raihn – Dream Foundation

5:40

Wrap Up

Dean Suhr

5:45-6:30

free time

6:30

Hosted casual dinner

casual hosted dinner - at the hotel

9:00

MLD After Hours ... an informal time

646 - Parsippany Suite

Saturday, August 3rd

8:30am

Doors open - respite room opens

Buffet breakfast starts


9:00AM




MLD Foundation Update - recent activities, legislation, etc.


Dean Suhr, President & Co-Founder,
Teryn Suhr, Executive Director & Co-Founder, MLD Foundation

MLD Research & Therapy Updates ... the search for a MLD cure


9:30AM




Bone Marrow & Stem Cell Transplants

transplantation as a therapy and transplant in conjunction with other therapies

Enzyme Replacement Therapy ... Providing the ARSA enzyme

Gene Therapy ... Repairing the defective genes
* Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector
* Clinical trial update
* Intracerebral Gene Therapy

Substrate Reduction Therapy ... Reducing the sulfatide build up with small molecule glycan-targeted drugs

Other research, obstacles to approval


RESEARCH PANEL:

Dr. Paul Szabolcs, (live video) Children's Hospital Pittsburgh
Dr. Brett Crawford, (audio) Biomarin

Dr. Alessandra Biffi (live video feed), San Raffaele, Italy

Dr. Caroline Sevin
(video interview), Paris

Dr. Eric Crombez
, Shire, Lexington, MA

 

10:15
 MLD Family Photo & break


10:40


Continued discussion


all

11:45   Honoring Our MLD Angels
12:15  lunch

Practical Living with MLD ... the practical aspects of day to day life with MLD


1:15


Compassion Fatigue Syndrome
- a personal story


- Mike Parowski


1:45


Exploring Useful Tools - sharing of things that work


- see list of panelists below


2:45


Therapy Dog Experience


- Stephanie Fitzhenry


3:00 break


3:15


Quality of Life on the MLD Journey

A casual, free-flowing, interactive discussion between attending families and the panelists.


PANEL:
- Dr. Julie Hauer, Pediatric Palliative & Pain Care Consultant, Boston, MA
- Dr. Lawrence Charnas
- Trudy Bier
, Physical Therapist
- Brittany Arrington
, Speech Language Pathologist/AAC Specialist
- Susan Lipe
, MLD mom

- and all of the families

5:10-5:15

Call to Action - how we can help each other and accelerate a cure

all

5:15

"Beating" MLD - a family activity you won't want to miss


6:30


Hosted Group Dinner

(carpools leave at 6:30, dinner starts at 7:00)

Black River Barn
1178 New Jersey 10, Randolph, NJ 07869
(973) 598-9988
click for map

9:00

MLD After Hours ... an informal time

646 - Parsippany Suite


* tbd - to be determined


Sunday

Sunday is an optional social day.
Hotel checkout is at noon
.


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

Shire Pharmaceuticals   Dylan's Dinosaur Stomp   Honoring Olivia   Eli & Ella's Prayer Warriors   Stennis Foundation   In Memory of Lucas Viscomi  
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.


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


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