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Muscular Dystrophy News
3 天
DMD treatment SMT-M01 gets FDA orphan drug, rare disease tags
DMD treatment SMT-01, a cell replacement therapy, received FDA orphan drug and rare pediatric disease designations.
Muscular Dystrophy News
4 天
Overcoming mental hurdles as I transition to using a walker
For a long time, columnist Robin Stemple resisted using a walker to navigate around his home, but eventually, he had to put ...
Muscular Dystrophy News
6 天
Losmapimod fails to significantly improve FSHD outcomes: Study
Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at ...
Muscular Dystrophy News
2 天
Going gray prompts questions about the impact of caregiving on my life
Columnist Betty Vertin is going gray — as in, she's decided to stop coloring her hair. Did DMD play a role in this decision, she wonders.
Muscular Dystrophy News
5 天
Why patient advocacy is important for those of us with Duchenne
Last Saturday, I had the honor of participating in the third Singapore Health Patient Advocate Connection event (SPACe) ...
Muscular Dystrophy News
12 天
Atamyo, Dion Foundation partner to expand LGMD study into US
Atamyo has partnered with the Dion Foundation to expand into the U.S. a clinical trial testing the gene therapy ATA-200 for ...
Muscular Dystrophy News
10 天
NS Pharma DMD treatment gets FDA rare pediatric disease status
The U.S. Food and Drug Administration granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping DMD ...
Muscular Dystrophy News
13 天
How my late brother and I left a mark on our DMD community
Columnist Shalom Lim reflects on how he and his late brother, Isaac, left their mark on the Singaporean DMD community as ...
Muscular Dystrophy News
10 天
Esteban Dominguez Cerezo, MS, Science Writer
The U.S. Food and Drug Administration has granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping Duchenne muscular dystrophy (DMD) treatment from NS Pharma. Treatments granted ...
Muscular Dystrophy News
13 天
Teaching students about life with limb-girdle muscular dystrophy
For columnist Patrick Moeschen, a teacher and wheelchair user, each school year is an opportunity for teaching students about ...
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