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Register for the Valley Forge MLD Family Conference™

Online Registration closes July 13th.
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2010 MLD Family Conference™ AGENDA

Sponsored by the MLD Foundation

August 6th-7th, 2010 in Cleveland, Ohio

as of August 1st, 2010 - subject to change (click here to view last year's agenda)

Friday August 6th


Doors open - sign in & registration, respite room opens

Hilton Garden Inn, Mayfield Village - Cleveland, Ohio


Welcome & Introductions

Dean Suhr
President, MLD Foundation

MLD 101 ... an overview of Metachromatic Leukodystrophy




What is MLD?

  - Neurology
  - Genetics
  - Testing - diagnosis, mutations

Newborn Screening for Lysosomal Diseases ...
Krabbe disease: the NYS model

Preimplantation Genetic Diagnosis

Dr. Lawrence Charnas - Pediatric Neurologist, University of Minnesota

Dr. Patricia Duffner, Professor of Neurology & Pediatrics, University of Buffalo School of Medicine; Clinical Director - Hunter James Kelly Research Institute,

Dr. Mark Hughes, Molecular Biochemist, Director - Genesis Genetics, Detroit MI (via video)

3:10  break




Brains for Brain
- MLD Core Research Projects
Research into basic science and emerging therapies

Open discussion ... Questions and Answers

Make a Wish Foundation®

Dr. Maurizio Scarpa - University of Padova, Dept. of Paediatrics, Italy

Kelly Kleinschmidt, Executive Director - The Make-A-Wish Foundation® of Greater Ohio, Kentucky, and Indiana Northern Ohio Region (specfic presentor tbd)


Wrap Up

Dean Suhr


free time


Hosted casual dinner

Hilton Garden Inn

Saturday August 7th


Hotel breakfast buffet (for hotel guests)



Doors open - respite room opens


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


Regulation, NIH/FDA

NIH Research initiative, New drug & therapy approval process, Orphan Drug Progam changes

Dean Suhr, President & Co-Founder, MLD Foundation


Bone Marrow & Stem Cell Transplants

transplantation as a therapy and transplant in conjunction with other therapies

Dr. Vinod K. Prasad
, MD, MRCP (London) Attending Physician Pediatric Bone Marrow and Stem Cell Transplant Service Duke University Medical Center, Durham NC

10:15  break



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

Enzyme Replacement Therapy ... Providing the ARSA enzyme
What is Enzyme Replacement Therapy?

Open discussion ... Questions and Answers
- Question about research underway
- Shire's HGT-1111 results & HGT-1110 plans

Dr. Maurizio Scarpa Dept. of Paediatrics - University of Padova, Italy

Dr. Marc Patterson
, Mayo Clinic, Rochester MN (via video)


12:00  lunch

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




- Why Advocate for your child?

- Developing a Home Care Plan for your MLD child

- Vignette #1: Seizures in MLD

- Vignette #2: Spasticity/Muscle Spasms in MLD

- Dr. Kendra Bjoraker, PhD Neuropsychology, Colorado Children's Hospital

- Dr. Julie Hauer, Pediatric Palliative & Pain Care Consultant, Boston, MA

- Dr. Lawrence Charnas - Pediatric Neurologist, University of Minnesota

- Dr. Lawrence Charnas - Pediatric Neurologist, University of Minnesota

3:00 break


Panel Discussion ...The MLD Journey

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

- Dr. Arthur Lavin, Pediatrician - Advanced Pediatrics, Cleveland OH
- Lauren Drobnjak, Physical Therapist - InvoHealthcare Assoc., Cleveland OH
- Dr. Neil Friedman M.B.Ch.B, Neurologist - Cleveland Clinic
- Jennifer Palmer RN - Pediatric Team Leader, Hospice of the Western Reserve
- Dr. Kendra Bjoraker, PhD Neuropsychology, Colorado Children's Hospital
- Dr. Julie Hauer, Pediatric Palliative & Pain Care Consultant, Boston, MA
- Dr. Lawrence Charnas - Pediatric Neurologist, University of Minnesota

and all of the families ...


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



free time


Hosted Group Dinner
(leave hotel at 6:15, dinner at 6:30)

Claddagh Irish Pub, Legacy Village, Lyndhurst, OH map to restaurant

* tba - to be announced

Sunday, August 9th

Sunday is an optional social day.
Buffet breakfast is provided for hotel guests. Hotel checkout is at noon

Thank you to our sponsors, service, & grant providers

Shire HGT   Mac's Team    The Stennis Foundation  

Shire HGT, Mac's Team, The Stennis Foundation, Olivia's Walk, and Boinx Software 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.

Contact Dean Suhr <> +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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