Exon Skipping Therapies News, Researches and Articles

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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

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

Editorials April 18, 2025 0

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)

Press Releases April 18, 2025 0

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

Editorials April 16, 2025 0

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

Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Treatments December 26, 2024 5

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

What is Duvyzat (Givinostat)?

Approved Treatments October 13, 2024 1

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

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

Editorials January 3, 2025 0

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

Exon Skipping Therapies

Must Read

ITF Therapeutics LLC Presents Long-Term Data of DUVYZAT™ (givinostat) for Treatment of Duchenne Muscular Dystrophy at MDA Clinical and Scientific Conference

Avidity Biosciences Announces Positive Topline Del-zota Data Showing Consistent, Statistically Significant Improvements in Dystrophin

Roche to Share Ongoing Work on Elevidys Gene Therapy at Muscular Dystrophy Association (MDA) 2025 Conference