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Latest Updates in SMA Translational Research
When: Saturday, April 5, 2014
Where: The Joseph B. Martin Conference Center at Harvard Medical School, 77 Avenue Louis Pasteur, Boston, MA 02115
Time: 8 AM – 1 PM
Light breakfast and lunch will be provided
Ahoy There Mateys! This year’s child entertainment will be PIRATED THEMED!
All ye Pirates shake with cheer as Boston SMA Day draws near!
Put on your eye patches and bring yer parrots.
As Joe the Storyteller describes all about it.
A Bearded Pirate may come along
So bring your best swash buckling song
You’ll walk the plank if you dare
Or under a balloon limbo pole with flair
Careful where you put your gold
Because at Boston SMA there is much to behold!
Register for event by March 24th at: http://bostonchildrens.org/smadayregistration
For questions, please contact Nicole Visyak: Nicole.email@example.com or 857-218-4677
All sessions open to researchers and families
8:00 - 8:30 AM
Registration and Refreshments
8:30 - 8:40 AM
Welcome and Introduction
8:40 - 9:10 AM
Keynote Speaker: Karen Chen- SMA Foundation
“An Introduction to Drug Development in SMA”
9:10 - 9:30 AM
Basil Darras- Boston Children’s Hospital
“Clinical Trials in Spinal Muscular Atrophy: An Update”
9:30 - 9:50 AM
Amy Pasternak- Boston Children’s Hospital
"Evidence- Based Outcome Measures in SMA”
9:50 - 10:00 AM
10:00 - 10:20 AM
Anne Hart - Brown University
“Delineation of cellular and genetic mechanisms underlying SMA”
10:20 - 10:40 AM
Shi Yan Ng- Harvard Stem Cell Institute
“Targeting the endoplasmic reticulum stress pathway in SMA motor neurons”
10:40 - 11:00 AM
Mustafa Sahin- Boston Children’s Hospital
"MiRNAs and mTOR in SMA”
11:00 - 11:10 AM
11:10 - 11:30 AM
Omar Khwaja- Roche Pharmaceuticals
“RG7800: a new molecule to treat SMA by altering SMN splicing”
11:30 - 11:50 AM
John Cherry - Pfizer
"Use of droplet diginal PCR to determine SMN copy number variation and quantify SMN mRNA expression”
11:50 - 12:10 PM
Kathy Klinger- Genzyme
12:10 - 12:30 PM
Helena Liedtke- Parent Perspective Lecture
“Experiences of child’s participation in ISIS Clinical Trials”
12:30 - 1:00 PM
Conclusions and Group Lunch
To park at the Joseph B. Martin Conference Center garage please follow the directions below:
If you are coming from Longwood Avenue:
If you are coming from Fenway:
If you are using a GPS system, the garage is directly underneath the Conference Center and thus has the same address:
77 Avenue Louis Pasteur, Boston, MA 02115
If at any time you find yourself lost or need assistance please call our main line at 617-432-8990.
The future of pediatrics will be forged by thinking differently, breaking paradigms and joining together in a shared vision of tackling the toughest challenges before us.”