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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 […]
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Losmapimod fails to significantly improve FSHD outcomes: Study
Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at improving […]
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Navigating Access & Resources
Navigating Access & Resources Navigating insurance access for medical care such as approved therapies or […]
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A friend in the DMD world makes a difference in my self-care
In my recent columns, I’ve shared several challenges we’ve faced in the past months of […]
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Atamyo, Dion Foundation partner to expand LGMD study into US
Atamyo Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand […]
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NS Pharma DMD treatment gets FDA rare pediatric disease status
The U.S. Food and Drug Administration has granted rare pediatric disease designation to NS-050/NCNP-03, an […]
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Watch the Recording: Advocating for A Successful School Year
Recently, PPMD’s Alexis Hazlett was joined by panelists Jodi Krause (Colorado Children’s Hospital), Jill Castle, […]
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How my late brother and I left a mark on our DMD community
While observing World Duchenne Awareness Day over the weekend, I reflected on the journey my […]
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Teaching students about life with limb-girdle muscular dystrophy
As most of America’s children go back to school, I thought it’d be timely to […]
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DYNE-251 leads to improvements in motor function in DMD: Update
Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led […]
