BLA 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

Givinostat EMA Approval: When Will Givinostat (Duvyzat) Be Approved in Europe?

Editorials April 14, 2025 0

Givinostat has already received regulatory approval in the US...

Satellos SAT-3247 Stem Cell Approach May Restore Muscle Regeneration in Duchenne Muscular Dystrophy

Editorials April 12, 2025 0

Genetic interventions such as exon skipping or gene therapy...

New Drug Submission for AGAMREE (vamorolone) Accepted for Priority Review by Health Canada for the Treatment of Duchenne Muscular Dystrophy

Press Releases April 8, 2025 0

Catalyst Pharmaceuticals has announced that Health Canada has accepted...

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...

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Must Read

Dyne Therapeutics Announces Unprecedented and Sustained Functional Improvement Through 18 Months from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy

Researchers in Madrid Make Important Strides in the Cure of Duchenne Muscular Dystrophy with Nanoparticles

Metagenomi’s New CRISPR System (VK CAST) Enables Large Gene Integration