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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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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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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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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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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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Edgewise Therapeutics Reports Positive Results on Sevasemten Program
Edgewise Therapeutics, which received early funding from CureDuchenne, has reported positive results in its sevasemten […]
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Edgewise Therapeutics Shares Positive Results on Sevasemten Program for Becker and Duchenne
Edgewise Therapeutics, Inc., has announced positive results in the company’s sevasemten program for both Becker […]
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An odd dream made me wonder how my disability affects others
I’m an early riser. As I mentioned in a past column, I like to wake […]
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FDA advisory committee meeting for deramiocel in DMD set for July
The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting […]
