MLD Family Conference™ ... Tarrytown, New York - 2016


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Registration Closed
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  Online registration for this conference closed on Sunday, June 12. 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



2016 MLD Family Conference™ 2016 AGENDA

Sponsored by the MLD Foundation

July 15-16th in Tarrytown, New York

as of July 11th, 2016– subject to change ... See last year's agenda here


Friday, July 15th

9:30

Doors open - sign in & registration,
respite room opens

Doubletree Tarrytown, Tarrytown, New York
map iconclick for address & map to hotel

Respite: Tarrytown Room
Meeting: Westchester South

10:00

Welcome & Introductions

- Dean Suhrmember of one of the MLD Boards, President & co-Founder, MLD Foundation

- Teryn Suhrmember of one of the MLD Boards, Executive Director & co-Founder, MLD Foundation


Metachromatic Leukodystrophy: Overview,
Genetics, Newborn Screening, & Research
Moderator: Dean Suhr, President, MLD Foundation

10:45

Understanding MLD
- Chemistry, biology, genetics, progression

Diagnostics
- Diagnostics & Sequencing

Research Collaborations
- Genotype/phenotype correlations
- Natural History & OpenNHS
- GLIA - Global Leukodystrophy Initiative

Developing & Approving a Therapy
- What's involved pre-human
- Human studies
- Natural History Studies
- Therapy approval

PANEL:

- Alessandra Biffi MDmember of one of the MLD Boards, Director, Gene Therapy Program - Dana Farber/Boston Children's; Assoc. Prof. - Harvard Med., Boston MA

- Brett Crawford Ph.D., Staff Scientist, BioMarin, Novato CA

- Dr. Adeline Vandervermember of one of the MLD Boards – Children's National Hospital, Washington DC

12:00 Lunch
 

1:00

Newborn Screening
- Science: Updates on the development of a MLD screen
- MLD NBS Pilot Study
- Public Health Perspective
- Next steps towards Uniform MLD NBS ... and how you can help!
- NBS Policy: RUSP Roundtable Initiative

PANEL:

- Dean Suhrmember of one of the MLD Boards, MLD Foundation

- Teryn Suhrmember of one of the MLD Boards, MLD Foundation

- Prof. Michael Gelb PhD – Adjunct Professor of Biochemistry, Univ. of Washington

2:15

Patient Powered Research
- Advancing research & improving quality of care
- CENA, PCORI, GLIA
- Hands on with the MLD Registry

- Dean Suhrmember of one of the MLD Boards, MLD Foundation

- Dr. Adeline Vandervermember of one of the MLD Boards – Children's National Hospital, Washington DC

3:00
break
 

3:15

Current Research & Therapies


RESEARCH:
- Gene therapy - Taipei, Taiwan
- Receptor Mediated Transport
- Gene therapy - London, Canada
- Substrate Reduction Therapy (SRT)

CURRENT THERAPIES:
- Stem Cell Transplant

CLINICAL TRIAL UPDATES :
- Enzyme Replacement Therapy (ERT)
- Intracerebral Gene Therapy - Paris
- Gene Therapy With Transplant - Milano
- other research

- Allesandro Aiuti MD PhD – Director, Immunohematology Pediatric Unit
San Raffaele Hospital; Clinical research coordinator and Head of Unit
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milano Italy

- Alessandra Biffi MDmember of one of the MLD Boards, Director, Gene Therapy Program - Dana Farber/Boston Children's; Assoc. Prof. - Harvard Med., Boston MA

- Dr. Lawrence Charnasmember of one of the MLD Boards - Senior Translational Medicine Expert Musculoskeletal Diseases –  Novartis Institutes For Biomedical Research, Boston, MA- (via telephone)

- Brett Crawford Ph.D., Staff Scientist, BioMarin, Novato CA

- Derek Kelaita, (video interview) – Vice President, Business Development, Armagen, Calabasas CA

- Joanne Kurtzberg MD, (video interview) – Chief Scientific Officer, Robertson Clinical and Translational Cell Therapy Program (CT2), Duke University, Duke NC

- Margaret Wasilewski, MD, Shire Pharmaceuticals, Lexington MA

5:15

Recap & Next Steps

- Dean Suhrmember of one of the MLD Boards, President & co-Founder, MLD Foundation

5:30-7:00
free time
 

7:00

Hosted casual dinner

casual hosted dinner in the hotel atrium

9:00 - midnight

MLD After Hours ... an informal time

Fri: #226, Sat : #452
Drop by whenever you can


tbd - to be determined

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



Saturday, July 16th


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

Moderator: Teryn Suhr, Executive Director, MLD Foundation



9:00am

9:15am
Respite room opens

Meeting room Doors open

Respite: Tarrytown Room

Meeting: Westchester South

9:30

MLD Care Issues


PANEL:

- Dr. Lawrence Charnasmember of one of the MLD Boards - Senior Translational Medicine Expert Musculoskeletal Diseases –  Novartis Institutes For Biomedical Research, Boston, MA

- Dr. Julie Hauermember of one of the MLD Boards, Pediatric Palliative & Pain Care Consultant, Boston Children's Hospital, Boston, MA

- Tara West, RN, MSN, CPNP (Certified Nurse Practitioner), Robertson Clinical and Translational Cell Therapy Program , Duke University, Durham NC

... and all of the families

10:30  break
10:45 -11:30 Accessing Services

ROUNDTABLE DISCUSSION:

- Tara West, RN, MSN, CPNP (Certified Nurse Practitioner), Robertson Clinical and Translational Cell Therapy Program , Duke University, Durham NC

... with all of the families

11:45 Group Picture location to be announced
12:00 Lunch  
1:00 MLD Angel Butterfly Ceremony
This will be live webcasted
everyone - location TBD

1:45

CBD Oil / Medical Cannabis

- What is Medical Cannabis?
- CBD vs. CBD with THC
- Legal, access, & regulatory issues
- Choices ... dose, strain, quality, & consistency
- How to measure and respond/adjust
- How do we share what we learn with others?

ROUNDTABLE DISCUSSION:

- Stephen Dahmer MD, Chief Medical Officer  – Vireo Health

... with all of the families

2:30

Alternative Therapies

ROUNDTABLE DISCUSSION:

- Shannon Moy, LAc, MAOM, Licensed Acupuncturist & Herbal Medicine Practitioner – Serenity Acupuncture, Golden Valley MN


... with all of the families

3:15 Break  
3:30 Essential Oils

ROUNDTABLE DISCUSSION:

- Adrian Moy AA, Certified Aromatherapist, Golden Valley MN


... with all of the families


4:15

Communication Challenges With MLD
How to Talk about MLD, Our Loved Ones, and Ourselves...

ROUNDTABLE DISCUSSION:

- Brett & Heidi Robertson, MLD parents

- Tara West, RN, MSN, CPNP (Certified Nurse Practitioner), Robertson Clinical and Translational Cell Therapy Program , Duke University, Durham NC


... with all of the families


5:15

Closing remarks, Group Hug

- Dean Suhrmember of one of the MLD Boards, President & co-Founder, MLD Foundation

- Teryn Suhrmember of one of the MLD Boards, Executive Director & co-Founder, MLD Foundation

5:30 - 7:00

free time

 

7:00

Celebrating MLD Foundation and the MLD Family™!
The first 30-45 minutes will be live webcasted including the parade MLD loved ones and the acknowledgement of 15 years.


Doubletree Tarrytown

11:00 -1:00

MLD After Hours ... an informal time

Fri: #226, Sat : #452
Drop by whenever you can


tbd - to be determined

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



Sunday, July 17th

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


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

Shire Pharmaceuticals   Believing For Bryleigh   Shire Pharmaceuticals  
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 organized by the MLD Foundation. Check with your tax advisor about the deductibility of any expenses associated with this conference.

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Lawrence Charnas, MD, PhD works in the Musculoskeletal Disease Translational Medicine group at Novartis Institutes of Biomedical Research (NIBR) in Cambridge, MA. He was previously Director & Head of Discovery Medicine at Shire in Lexington, Massachusetts where he was the Medical lead on several clinical development programs including ACE031, a soluble ActRIIb receptor for Duchenne Muscular Dystrophy and an enzyme replacement therapy in Globoid Cell Leukodystrophy (Krabbe Disease).

Prior to joining Shire, he was an Associate Professor of Pediatrics and Neurology at University of Minnesota, working on the treatment of Adrenoleukodystrophy and other lysosomal leukodystrophies with Hematopoietic Cell Transplant. Prior to joining the faculty at University of Minnesota, he had developed expertise in a number of other rare, pediatric genetic diseases including cystinosis, osteogenesis imperfecta and the Oculocerebrorenal syndrome of Lowe.

He received his B.A. in Chemistry (Cornell University, Ithaca, NY), an MD and PhD - Genetics ( University of Pennsylvania, Philadelphia, PA) and clinical training in Adult Neurology (Johns Hopkins Hospital, Baltimore, MD), Medical and Biochemical Genetics (NICHD, Bethesda, MD), and Pediatric Neurology (University of Minnesota).

He has co-authored many peer reviewed articles, and serves on the Medical and Scientific Advisory Boards of the MLD Foundation and Lowe Syndrome Association.

Dr. Wenger directs the largest lysosomal storage diseases (LSD) testing laboratory in the world at Jefferson University in Philadelphia. MLD Foundation considers his work to be the gold standard of consistency and quality for MLD and other lysosomal disease (LSD) diagnostics.

Dr. Wenger is a member of the MLD Foundation Medical and Scientific Advisory Board.

The LSDs are a group of genetic disorders cause by mutations in genes that code for proteins required for the lysosomal catabolism of naturally occurring carbohydrates, proteins, and lipids. About 35 such disorders have been defined and over 20 are diagnosed in this laboratory. Samples are sent to this laboratory from around the world for help in diagnosing a patient with certain symptoms, for carrier testing and for prenatal testing. Most diagnostic tests are enzyme-based but others require the use of specific antibodies, tissue extraction, radio-labelled lipid loading or molecular analysis for known mutations. The experience of the research laboratory is brought to diagnostic laboratory in the form of new test development, and interesting patients can be studied in the research laboratory to help answer important questions regarding protein structure and function.

Medical School
BS, Temple University - School of Pharmacy, Pharmacy - 1964
PhD, Temple University - School of Medicine, Biochemistry - 1968

Board Certification
Licensure, CLIA,
American College of Medical Genetics, American Board of Medical Genetics, Board Certification, Clinical Biochemical Genetics and Clinical Molecular Genetics

Research and Clinical Interests
Krabbe disease ... Dr. Wenger's laboratory the first laboratory to purify GALC and clone the GALC cDNA and gene which causes KD. The lab has diagnosed nearly 400 patients of all ages with KD spanning nearly 60 mutations. Dr. Wenger has placed human and mouse cDNAs into several viral vectors including retroviral, adeno-associated viruses, lentivirus and SV40. They have successfully transduced oligodendrocytes from the twitcher mice model with viral vectors containing human GALC cDNA and corrected them to a normal phenotype.

ACADEMIC APPOINTMENTS (1997-present):

Jefferson Medical College, Philadelphia, PA. September 1986 - November 1998, Professor of Medicine (Medical Genetics), and Biochemistry and Molecular Pharmacology

Director, Lysosomal Diseases Testing Laboratory. 1974-present

OTHER ACTIVITIES (1997-present)

Scientific Advisory Committee of the National Tay-Sachs and Allied Diseases Association

Advisor - United Leukodystrophy Foundation, Inc.

Editorial Board of Molecular Genetics and Metabolism (1991-present)

Medical and Scientific Advisory Board - MLD Foundation

Scientific Advisory Board of the National Mucopolysaccharidosis Society

Scientific/Medical Advisory Committee of the Canavan Disease Foundation

Program Committee-American Society of Human Genetics (1994-1997)

NIH Board of Scientific Counselors; review of Intramural Research Programs at NIH, October 1997

Working Group of NIGMS Human Genetic Cell Repository, 2001-2004