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Muscular Dystrophy News
11 小时
EMA agency continues to oppose renewing Translarna’s approval
CHMP upheld earlier opinions that Translarna is not of significant benefit to DMD due to nonsense mutations; European ...
Muscular Dystrophy News
1 天
Changing society and its views on disability, one column at a time
So we have to change society. It’s a tall order, but it’s been done over and over again throughout history. It starts with a ...
Muscular Dystrophy News
4 天
I wish all of my sons could have played football
Columnist Betty Vertin, watching her son Chance complete his final season, wishes her sons with DMD could've played football, too.
Muscular Dystrophy News
5 天
RNA-based approach shows promise for DM1 in cell models
Treatment with an RNA-based therapy approach was found to correct molecular defects in a cell model of myotonic dystrophy ...
Muscular Dystrophy News
6 天
An emergency preparedness plan is crucial for those with disabilities
Hurricanes in the southeastern U.S. prompt columnist Robin Stemple, who has FSHD, to consider his emergency preparedness plan ...
Muscular Dystrophy News
7 天
Gene therapy at low dose helps two OPMD patients with swallowing
A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with oculopharyngeal muscular dystrophy (OPMD) treated in a Phase 1b/2a clinical trial, ...
Muscular Dystrophy News
11 天
Every week, we try to organize chaos in our family of 9
Columnist Betty Vertin, who has seven children, tries to organize the chaos in her family's lives, though no week is typical.
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