Precision BioSciences Highlights New Preclinical Data for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy

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Precision BioSciences a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene...

Vyondys 53: What Does FDA Rejection Letter Mean?

Editorials DMD Warrior -
The Food and Drug Administration published more than 200 letters it sent to companies after their medications were rejected...

Sarepta Therapeutics Halts ELEVIDYS Shipments for Non-Ambulatory DMD Patients

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Due to two documented incidents of severe liver failure that resulted in death, Sarepta Therapeutics has stopped shipping its...
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We do not want children to die from a disease that can be cured. We want to give hope to families and ensure that all children with DMD are treated and regain their health.

Latest Articles

Precision BioSciences Highlights New Preclinical Data for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy

Press Releases 0
Precision BioSciences a clinical stage gene editing company utilizing...

Vyondys 53: What Does FDA Rejection Letter Mean?

Editorials 0
The Food and Drug Administration published more than 200...

Sarepta Therapeutics Halts ELEVIDYS Shipments for Non-Ambulatory DMD Patients

Press Releases 0
Due to two documented incidents of severe liver failure...

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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Treatments 5
A genetic condition known as Duchenne muscular dystrophy (DMD)...

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Research 5
The results of new gene therapy studies for Duchenne...

Duchenne Muscular Dystrophy: Treatment & Cost

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The excitement you feel until your child is born...

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