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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 […]
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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 […]
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Regulatory Update on Capricor’s Deramiocel for Duchenne
Capricor Therapeutics, which received early funding from CureDuchenne, has indicated that the FDA has decided […]
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Capricor Shares Regulatory Update on Deramiocel BLA for Duchenne
Capricor Therapeutics has shared a regulatory update related to its Biologics License Application (BLA) for […]
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MDA Ambassador Guest Blog: Finding Passion & Purpose in Art
Jennifer Lane is a wife and mother of three grown children and lives in Alabama. […]
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Your Guide to Genetic Testing for Neuromuscular Diseases
Genetic testing is an important part of diagnosing and managing neuromuscular diseases, including muscular dystrophies, […]
