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Webinar Recording: Edgewise Therapeutics – Update on Sevasemten Program in Becker and Duchenne
Edgewise Therapeutics recently joined PPMD for a community webinar for an update on the company’s
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Ifetroban continues to show cardiac protection in Phase 2 DMD trial
Ifetroban, Cumberland Pharmaceuticals’ investigational oral therapy, improved heart function and reduced cardiac damage biomarkers in
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Craft Your Story
Crafting Your Story for Awareness, Connection, and Change Webinar. Watch HERE The post Craft Your
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Clinical Research Alert: Phase 3 Study of RGX-202 in Boys with DMD
REGENXBIO Inc. is enrolling boys living with Duchenne muscular dystrophy (DMD) to participate in a
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Simply Stated: MG Therapeutic Landscape
Myasthenia gravis (MG) is a chronic neuromuscular disease characterized by muscle weakness that worsens after
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Arizona Becomes the Latest State to Require Newborn Screening for Duchenne
We are proud to share some important news for our community: Arizona has officially joined
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Accepting help is difficult for me as a mom and a caregiver
Late afternoon sunlight drifts into the house, brightening the blue paint on the walls. The
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Precision’s gene-editing therapy gets FDA’s rare pediatric disease status
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD,
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MDA Ambassadors Share Tips for Flying with Power Wheelchairs and Medical Equipment
Air travel, even in the best circumstances, can be a little stressful. Flying with a
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Edgewise Therapeutics Reports Positive Results on Sevasemten Program
Edgewise Therapeutics, which received early funding from CureDuchenne, has reported positive results in its sevasemten
