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CRISPR-Cas9

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Del-zota (EXPLORE44) Provides Nearly 30% Normal Dystrophin Production in Patients Amenable to Exon 44 Skipping

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A phase 1/2 EXPLORE44 study (NCT05670730) is now being...

Belief BioMed BBM-D101 Gene Therapy Receives IND Clearance by National Medical Products Administration (NMPA)

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Belief BioMed (BBM), an innovative biotechnology company focused on...

Dr. Aravindhan Veerapandiyan, a neurologist at Arkansas Children’s Hospital, Reviews RGX-202 Gene Therapy Study

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At the American Academy of Neurology Annual Meeting, which...

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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)...

What is Duvyzat (Givinostat)?

Approved Treatments 1
For individuals with Duchenne muscular dystrophy (DMD), adults and...

In Which Countries Is Elevidys Approved? Is Elevidys Approved In Europe?

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Elevidys is the only approved gene therapy in the...

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