Orphan Drug 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

The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys

Press Releases July 25, 2025 0

Roche announced today that the Committee for Medicinal Products...

Brazil Suspends Use of ELEVIDYS (delandistrogene moxeparvovec)

Press Releases July 25, 2025 0

The Brazilian Health Regulatory Agency (Anvisa) announced that it...

Mutations and Deletions Amenable to Exon 44 Skipping Therapies for Duchenne Muscular Dystrophy

DMD July 24, 2025 0

Exon 44 skipping has emerged as a targeted treatment...

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)...

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Research January 5, 2025 5

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

Duchenne Muscular Dystrophy: Treatment & Cost

Editorials January 2, 2025 1

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

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Must Read

Entrada Therapeutics Receives EU Authorization to Start Exon 45 Skipping Clinical Trial in Duchenne Muscular Dystrophy

Entos Pharmaceuticals Develops Re-Dosable, Full-Length Dystrophin Gene Therapy: Fusogenix PLV Method

Genethon Reports Results of GNT0004 Gene Therapy Clinical Trial: Permanent Decrease in CK Levels of More Than 75%