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Publications >
QUEST Vol.16 No.1 January-March 2009
Check out the complete online version of Quest!
QUEST
Volume 16, Number 1
January - March 2009
COVER
Gaining Their Sea Legs
Adventure awaits when a family of 11 — including four boys with Duchenne muscular dystrophy — takes to the sea for a year.
FEATURES
DEPARTMENTS
Mattie’s Park
A wonderful tribute to a former MDA National Goodwill Ambassador is now reality.
Rescued Lives
Myozyme answers an SOS from the Pompe community — but responses to the drug vary, cost is high, and some questions remain unanswered.
Stand Up!
The benefits of standers for children are clear.
Focus On: Drug Development Progress
A host of projects are in the pipeline for DMD, SMA and FA — see where they all stand.
Billy & the Kid
2009 ushers in role changes for MDA’s Dynamic Duo: Billy Gilman and Luke Christie.
Writing Off Caregiver Expenses
ADA Violations in Medicaid Cuts
Attorney Steve Gold warns advocates to keep an eye on their state legislatures.
Breathe Deeply and Let Your Baby Go to Camp
A primer for nervous parents
Keeping in Touch
MDA Mailbag
MDA Matters
Research Updates
As the Wheel Turns
Wheelchair accessories
MDA Advocacy Report
To Boldly Go
Accessible Japan
Quest Tech
Funding communication
Insider
Rolling with Laughter
S’up neighbor?
Product Peeks
InfoQuest
Quest Magazine Online
New in 2009!
From Where I Sit
The meaning of ‘can’t’
Research Updates
Blood-vessel narrowing implicated in exercise-related fatigue
Lab-engineered protein increased utrophin production in DMD mice
Sarcospan protein unexpectedly saves MD-affected muscle fibers
Signal-blocking compound markedly improved heart function in EDMD mice
AAV9 achieves body-wide gene delivery
Neural stem cells aid survival and function in SMA mice
Missing link identified at nerve-muscle junction
DNA test now available based on MDA grantee’s ID of gene for CNM
Research Adiministration
Updated MD-CARE Act is now law
Boston Biomedical becomes Wellstone MD center
MDA grantee Ronald Cohn receives award
Clinical Trials and Studies
Genzyme continues studying Myozyme in Pompe disease
Amicus is studying pharmacological chaperone in Pompe disease
Diaminopyridine under study in Lambert-Eaton syndrome
Acceleron testing compound to slow muscle wasting
Exon skipping drug may get EU orphan drug status
Study of thymectomy in people with MG broadens criteria
Researchers are seeking metabolic ‘signature’ of mitochondrial diseases
Study of exercise in mitochondrial disease under way at UT Southwestern
Graduate student seeks parents’ views of genetic counseling
for DMD, BMD
SMA registry now allows Web-based participation
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